The Big Red Biotech Blog

Senators Ask FTC to Probe KV For Price Gouging

KV has stirred a hornet's nest among various patient, physician and thrid party payer constituencies with its pricing decision on the its new orphan drug Makena. Now its has the Senate and possibly the FTC to contend with.

Senators Ask FTC To Probe KV For Price Gouging // Pharmalot.

Readers will note that KV, upon FDA-approval of its version of the drug last week, chose to raise the price from about $20-$30 per dose received from most pharmacies to a new $1500 per dose price.  This has raised hackles amongst almost everyone execept perhaps KV investors.

Two senators - Minnesota Democrat Amy Klobuchar and Ohio Democrat Sherrod Brown - wrote FTC chair Jon Leibowitz that KV may be engaging in anti-competitive behavior. They maintain that a KV patient assistance program is insufficient because it does not cover “certain groups” of women.

Further, the Senaotrs said KV Pharmaceutical is taking advantage of FDA’s approval of Makena and orphan drug status leading to a monopolization of treatments to address preterm labors. The price increase will place a heavy burden on state Medicaid programs and may result in diminished access to appropriate health care for women and in increased preterm births.

"It’s critical that we make sure this company isn’t taking advantage of its orphan-drug determination to monopolize the market and engage in price gouging at the expense of pregnant women,” Klobuchar says.

This would appear to be an example of an unintended consequence of the orphan drug laws which primarily were meant to provide manufacturers incentives to bring new drugs to market for conditions with unmet need -- and for which the economic incentives to do so needed a boost.  What's happening here is that KV has identified a drug which has been around for more than 50 years -- but has never gone through an FDA approval process. 

KV recognized this and created an approved drug - which may in fact have some enhanced quality aspects.  However, KV has errored in my view in raising its price so egregiously - it appears greedy.  Maybe this is from need -- as it is trying to recover from a consent decree for distributing adulterated and unapproved drugs -- although that might be viewed as troubling in an of itself in terms of a pattern of corporate behavior.  Regardless, the pricing decision will likely encourage the congress in the time of budget crisis -- particularly around healthcare -- to review how it has structured orphan status for long known but unapproved drugs.  I would expect some type of redress but we'll see.

 
Posted by Bruce Lehr March 19th 2011.

Fabry Patients Denied March-In Now Sue Under Bayh-Dole

As reported in the Pharmalot blog and the Patent Doc blog, six Fabry patients are now suing Genzyme and Mt. Sinai hospital as individuals and as a class for their failure to provide Fabrazyme to treat their disease.   Previously these patients had asked HHS to exercise march-in rights to find another manufacturer for the product and they have also sued to force thr rationed drug to be sold in the US first before being shipped overseas as the inventions allowing the drug to be made were paid for by US tax dollars. 

C. Allen Black, Jr., who represents the Fabry patients in this case, said in a press release that "for the first time, the courts will be asked to determine whether patients who are guaranteed access to tax-payer funded inventions under the Bayh-Dole act may recover individual damages under the statute when a contractor misuses the invention or withholds it from the public."

Black states further, “It defies belief that, even though US tax dollars paid for the invention of the drug and its development, American citizens are now told that they must suffer and die while paying almost $240,000 a year for a dose of drug that is untested, dangerous, and too low to even help them. Worse, some of my clients were diagnosed after June 2009, so Genzyme simply bans those Americans from receiving any drug at all.”

Fabry patients have requested compensation and punitive damages for lack of adequate treatment and Genzyme's failure to take adequate precautions including keeping a reserve inventory and providing second sources of manufacturing.

Sanofi's Rare Disease Competitors Welcome Cash Influx

Xconomy writes that smaller competitors in rare disease niche's are on balance happy about Sanofi's decision to buy Genzyme for its presence in this area of therapeutics. They believe that more investment in the area is good for everyone - patients and drug company competitors alike as it brings more attention and investment. 

I found it interesting that there didn't seem to be too much fear (expressed in the article anyway) that smaller competitors would have trouble competing versus Sanofi's bigger pockets.  Quite the contrary, Shire's spokesman expressed the opinion that the whole area woudl benefit from increased investment.  What's more?  They didn't seem intimidated at all on the prospect of competing for deals with its richer new big brother in the field.

The Shire spokesman opined that smaller companies like itself were often more flexible in negotiating terms that were fairer and were more open to forging alliances.  They didn't feel a need to force a particular deal structure -- and also felt they could be more nimble in evaluating deals and moving to close them quicker. 

This brought to mind a couple of thoughts.  One - just look at how long Sanofi and Genzyme danced around before this deal closed.  Two - Kevin Kinsella's recent comments regarding predatory big pharma tactics.  Will these work in an environment where competition for the deal exists and where one party is willing to be more flexible on its terms?  Maybe Shire has something here. Bring on the money and the interest.

 

 

 

 

Posted by Bruce Lehr March 3rd 2011.

Traditional Drug Model Overripe?

Here's another article, this time from NatureNews, claiming the current Big Pharma drug discovery model is past its time and will need to be replaced by something new - in this case with a more "open innovation" model consisting of participants from industry, academia, government and even charities.  Not a new idea but seemingly a persistent one even in face of historic opposition.

Clearly, the industry is reshaping R&D.  We've seen large cuts in Big Pharma R&D.  We're starting to see outsourcing.  We're seeing venture capital funding.  We're seeing technology and company acquisitions. Are we seeing open innovation?  Well yes, in the case of rare diseases -- and possibly for diseases endemic to only developing markets where the tradtional economic model looks weak.

An open innovation model is scientifically appealing. One problem it addresses well is the tendency of drug companies to pursue similar or identical target molecules for certain diseases.  It would be beneficial to know in advance that a certain approach has already been tried and failed - rather than throw away the time and effort to repeat a failed clinical study.  Not to mention sparing clinical trial participants the exposure to a failed approach.

But this type of model requires a certain amount of cooperation on the IP front.  Information needs to be shared at early stages in development. The model would also likely involve academic institutions in these early stages and these groups need to be funded. That may require cooperation from both the pharma industry, government and the aforementioned charitable funding agencies to provide the bucks to complete the work.

The carrot for industry in this is that it would share risk and cut expense for them.  But, industry still wants a return on whatever funding it provides -- unlike perhaps a charitable agency or a government which may be more satisfied with the cure itself regardless of whether it collects direct profits. Industry representatives who have commented on this approach have noted that IP concerns remain the biggest hurdle for them in investing in this model.  This model will likely get a trial in malaria.

But, industry being industry wisely will not put all its eggs in one basket.  Other academic-industry models are being employed where individual pharma companies have provided funding to academic partners -- usually in targeted disease areas -- in return for right of first refusal on discoveries. The idea is to make the academic partners into more of the industrial discovery arm. And this model, does take care of industry's IP concerns.

At least people are talking about different models now as the old standbys do appear to have run their effective course.  We will need to see which models apply best in certain situations.  It is likely better to have several working and competing models that can be applied to the proper circumstances.  One size is unlikley to fit all.

Posted by Bruce Lehr March 2nd 2011.

 

 

Amicus Moves In on Rare Disease Market

Amicus Therapeutics believes that it has the opportunity to make a move in the rare disease market - competing with companies like Genzyme for Fabry's Disease patients as an example. This year, Amicus' Amigal was able to capture market share during Genzyme's supply problems with its drug Fabrazyme.

Amicus CEO, John Crowley says "It is good for people to have options for the treatment of rare diseases."  See Fierce Biotech.

 

 

Posted by Bruce Lehr Feb 16th 2011.

Optimize Neglected Disease Funding Mix

 This Reuters story covers new findings from the Global Funding of Innovation for Neglected Diseases (G-FINDER) report.  In it, the author's record that overall research funding went up by 8.2 percent to $3.2 B in 2009 but funding to important product development partnerships (PDPs) dropped.

PDPs are collaborations between drugmakers and research groups.  These have been successful in the past few years bringing an inexpensive meningitis A vaccine to market and there is another PDP produced tuberculosis vaccine in late stage testing.  The authors of the G-FINDER report say other similar projects could deliver many more useful products - compared to other research groups.

Both Jon Pender, of British drugmaker GlaxoSmithKline, and Joe Cerrell of the Bill & Melinda Gates Foundation which funded the report, said there was a "worrying trend" away from investment in PDPs, just when they need more money to further a potentially promising pipeline.  The report said such partnerships currently have around 140 drugs, vaccines and diagnostic tools in development, including a malaria vaccine from GSK which is in late-stage trials.

The potential impact of these products could be enormous in terms of public health in neglected regions. It is not just spending the money - but how it is spent.  And, these authors at least favor more, not less spending with PDPs.

Posted by Bruce Lehr Feb 16th 2011.

Everyone Wants to Adopt Orphan Drugs Now

From the Pharmalot, we have some interesting statistics regarding activity in the orphan drug arena.  As we know, The Orphan Drug Act encourages development of drugs for diseases with small markets.  It gives drugmakers seven years in which to sell a product without competition and sometimes tax incentives, too.

Orphan Drugs: More Designations, Fewer Approvals // Pharmalot.

With the advent of patent cliffs and revamping R&D groups in Big Pharma, as well as directional changes away from chasing so-called "blockbuster models", orphan and rare disease markets have become more in vogue. Several large pharma companies have launched specific initiatives in this sphere including Pfizer, GSK and Sanofi.

Now let's review the recent stats.  Last year, 323 requests went to the FDA for orphan status and 192 were awarded with that designation. There are now 2308 drugs designated as orphans. Designated n development and approved for sale are two different things it seems.  The FDA approved 14 orphan meds in 2010, 17 in 2009, which ar eboth below the peak year of 25 in 1996.

Based on the stats, a given Company's reach may exceed its grasp when pursing orphan drugs -- but the opportunity to get a lock on a market for several years is apparently irresistable at a time when FDA scrutiny of new drugs is particularly high and drug development is so expensive.  Of course, when everyone pursues the same strategy some are destined to be disappointed as well - and the stats would seem to reveal that news.

Posted by Bruce Lehr Feb 1st 2011.

Protalix Believes Oral Gaucher's Drug on Horizon

Protalix may be gaining on Genzyme in two treatment areas.

Protalix announced this week that it is making strong progress on the development of an oral drug for Gaucher's disease.  This would obviate the need for intravenous administrations (like Genzyme's current therapy Cerezyme).  Patients like oral administration much better which results in higher drug taking compliance and better disease management -- not to mention happier patients.

Similarly, Protalix reports making progress in one of its preclinical drugs - PRX-102 - for Fabry's disease. They tout this new drug as a potential best in class therapy.  Genzyme of course has struggled mightily in the past to years to make enough of its Fabrazyme - due to sever manufacturing issues that have resulted in it being under a FDA consent decree. 

Shire recently introduced a competitive therapy in Europe - Replagel.  And Fabry patients in the US have petitioned the NIH to give patent rights to another manufacturer to make Fabrazyme - and have petitioned the FDA to require Fabrazyme supplies to be sold in the US first before servicing Europe (as Europe has Replagel and the US does not).   The opportunity clearly exists for Protalix if its therapy holds up under clinical trials.  See Fierce Biotech.

"Don't look back [Genzyme], something might be gaining on you"  -- Satchel Paige

 

Posted by Bruce Lehr Jan 21st 2011.

Patients Petition FDA Over Genzyme Drug Shortage

Fabry patients in need of Genzyme's Fabrazyme are challenging again.  Last month, the challengers lost their attempt to get the NIH to exercise so-called march-in rights to allow another manufacturer to enter the market to make Fabrazyme - for which Genzyme hoolds the license to the patents.  This request was denied due to the NIH's feeling that no alternative manufacturer could start now and bring drug to market before Genzyme could get back into fuller production.

Patients Petition FDA Over Genzyme Drug Shortage // Pharmalot.

Now two of these patients are asking the FDA to regulate how Genzyme allots its drug.  It is asking the agency to make sure drug goes to the US market first before going to Europe -- apparently 62% of the drug is shipped overseas.  The EMEA has licensed an alternative drug, Replagal, made by Shire for the treatment of Fabry's.  The same drug is not licesned in the US and Shire as yet has not applied for one.

The US market is therefore short of Fabrazyme - where current patients subsist on a suboptimal dose in many cases and new patients can't get any drug - and has no access to Replagal.  There have been 3 reported deaths of Fabry's disease patients in the US due to the shortage.  No such deaths have been reported in Europe. Now the FDA is beginning to import Replagal to replace the Fabrazyme which was exported -- but patients must show "critical need" on a case by case basis to receive the US-unapproved drug.

Stay tuned for this one.

 

Posted by Bruce Lehr Jan 20th 2011.

Genzyme Builds new Myozyme/Lumizyme Plant in Belgium

Genzynme will spend Eu 250 M on a new manufacturing plant for its Pompe's disease drugs Myozyme and Lumizyme.  The plant will be located in Geel, Belgium and the company expects commercial regulatory approval of the site to be secured by 2014.

Genzyme to build New €250M Myozyme/Lumizyme Production Plant in Belgium.

The new Geel plant will have a maximum batch production capacity of 8000L and the existing Geel plant will add a thrid 12,000 L bioreactor. The expansion is meant to provide more capacity and better risk management for Genzyme. In recent years, the company has experienced well documented manufacturing failures (and is currently under a FDA consent  decree as a result) that have led to global drug shortages of its orphan products. 

Lumizyme and Myozyme are the only approved therapies for Pompe's disease at present.  Genzyme services more than 1400 of the estimated 10,000 patients with these afflctions. The commercial potential of these Pompe's disease drugs are expected to rival its best-selling Cerezyme for the treatment of Gaucher's disease. Genzyme is projects sales growth in excess of 30% with these products - especially if supplies are available for the full year in 2011. Sales wereup 39% ($128 M total) in the 4th quarter of 2010.

 

Posted by Bruce Lehr January 20th 2011.

NIH Denies Patient Bid To Break Genzyme Patent

From the Pharmalot blog, 3 patients are foiled in their attempt to obtain a new license from the NIH via a march-in petition to allow them to produce (seek another supplier) a competitive product to Genzyme's Fabrazyme to treat Fabry's disease. This after Genzyme has suffered manufacturing woes with the product for more than 1 year.

NIH Denies Patient Bid To Break Genzyme Patent // Pharmalot.

The Department of HHS denied the request saying this proposed solution would not redress the problem in the short-run. Also, no manufacturer has ever asked for a license besides Genzyme so HHS declined to grant one to the patients under these circumstances. HHS says it will seriously consider petitions by other qualified manufacturers.

 

Posted by Bruce Lehr December 7th 2010.

 

Orphan Drugs are Safe in the Shire

Shire's CEO, Angus Russell, says that he is comfortable that the company's niche in orphan drugs is a safe place.  Shire has grown the share of revenues that orphan drugs occupy in its own portfolio from less than 5% to more than 30% in the past three years.

Russell is confident in the orphan business model as an alternative to the blockbuster model (which seems to be coming to an end as an option) will sustain Shire.  This is true even if larger competitors like GSK or Pfizer enter the orphan space - or Sanofi is successful in buying Genzyme.  That's because orphan drug status confers market exclusivity - 10 years in the EU and Japan and 12 years in the USA. 

Shire has been able to establish a position in Fabry's disease with its drug, Replagal, and in Gaucher's disease with Vpriv - successfully competing against Genzyme's Fabrazyme and Cerezyme respectively.  Of course, this is largely due to Genzyme stubbing its toe in a major way with the manufacture of both their products - which opened the door for Shire's entrance to these patients.

Russell also thinks the Big Pharma competitors will need to learn how to compete in the orphan markets which is not like competing in the larger global markets with their large patient populations.  Russell notes that 'we work with a very tiny, discreet patient population."  Selling orphan drugs is a very patient intimate selling and servicing process.  See WSJ.

In that process, there is safety with the Shire.

 

Posted by Bruce Lehr November 22nd 2010.

 

GSK closes another "Rare Disease" deal with Amicus for Fabry's Disease

Previously, GSK announced its intention to focus on new treatments for up to 200 rare diseases. Last week, it announced agreements with Fondazione Telethon and Fondazione San Raffaele for a novel gene therapy treatment for ADA Severe Combined Immune Deficiency. 

Yesterday, GSK announced another deal with Amicus Therapeutics for its treatment, Amigal, for Fabry's Disease.  Fabry's is an inherited disease that affects between 5000 and 10,000 patients worldwide. Fabry's has been in the news a lot in the past year due to Genzyme's inability to fully supply its enzyme-replacement treatment for the disease because of manufacturing problems in its torubled Allston plant.

GSK will pay Amicus $60 M upfront and the deal included milestone payments up to another $170 M.  GSK also took the opportunity to bring its stock position with Amicus up to 19.9% ownership. Amigal is now in phase III trials and GSK gains global rights to develop, manufacture and distribute.  Amicus-GSK will explore combining Amigal with enzyme replacment therapy as part of their study.  Amigal represents a first in class attempt of an oral medication for Fabry's. See Fierce Biotech.

 

 

Posted by Bruce Lehr October 30th 2010.

GSK Also Attacks Rare Diseases

GSK amplified on its plans to focus on rare diseases as part of its strategic mission.  Their goal is to develop therapies for a list of around 200 rare diseases out of the estimated 6000-8000 known diseases. A GSK spokeman said  "We will target those diseases where we realistically believe we can make a significant contribution; ideally we will be creating new medicines, but importantly by adding to the weight of scientific knowledge into rare diseases to the benefit of all working in this area."  See also Bioreasearch Online.

As a first foray in the area, GSK announced a licensing agreement Monday with Fondazione Telethon and Fondazione San Raffaele for a novel gene therapy treatment for ADA Severe Combined Immune Deficiency (ADA-SCID) - a rare life-threatening immunodeficiency that affects 350 children worldwide.  Working with the same two partners, GSK will also develop six more ex vivo stem cell treatments for a range of disorders including - metachromatic leukodystrophy (MLD) and Wiskott-Aldrich Syndrome (WAS), beta-thalasemia, mucopolysaccharoides type I (MPS), globoid leukodystrophy (GLD) and chronic granulomatous disorder (CGD).  All of the disorders are caused by a single gene defect and have well understood molecular mechanisms.  Clinical trials for MLD and WAS are currently underway.

"This collaboration is a breakthrough for gene therapy in rare diseases," said Professor Luigi Naldini, Director of San Raffaele Telethon Insititute for Gene Therapy, "We have spent years researching these promising new technologiess, but without GSK's experience in developing medicines, we would not be able to take all the steps towards making a potential therapy available to patients."

I'm actually both stunned and impressed that GSK can pursue a strategy that is such a divers set of niche diseases and presumably do so profitably.  It would appear to be a Godsend to those patients with one of these very rare diseases that would certainly not otherwise be addressed in any large scale or systematic fashion.   

 

 

 

Posted by Bruce Lehr October 22nd 2010

Big Pharma and Governments Put Up $150 M to Fight Neglected Diseases

As reported in the Financial Times, six Big Pharma companies and several world goverments have pledged $150 M to fund a fight against 17 neglected diseases.  These include leprosy, lymphatic filariasis, and sleeping sickness which afflict more than 1 B people worldwide - often in the poorest of countries.  The funding plan and disease fighting effort was announced last Thursday by the World Health Organization (WHO).  WHO maintains that several of the 17 target diseases could be eliminated by 2020 simply by distributing existing medications more effectively.

Margaret Chan, head of the UN agency, said “If [preventitive chemotherapy with cocktails of cheap drugs] implemented widely, they can substantially reduce the disease burden, breaking a cycle of infection, disability and lost opportunities that keep people in poverty.”

The companies stepping up with funding are Novartis, GlaxoSmithKline, Sanofi-Aventis, Johnson & Johnson, Bayer and Eisai of Japan. 

  

   
 

Posted by Bruce Lehr Ocotber 17th 2010.

 

How To Speed Rare Disease R&D?

The Institute of Medicine (IOM) produced a new report replete with recommendations as to how to better harnass research and development efforts to attack rare diseases and develop more orphan therapies. Rare diseases are a problem as they affect millions. Unfortunately, those millions afflicted are dispersed among as many as 5000-8000 diseases - some afflicting only a few individuals and some affecting thousands.  I never thought about it before - but there may even be a pecking order within rare diseases themselves.

In general, traditional pharma companies have not attacked these diseases as it is very technically challenging and difficult to earn a return. Among the difficulties, one could count funding, access to adequate patient populations, clinical trial design and execution in small populations, etc.  With many of these diseases, not even the most basic mechanistic information about the disease is known.  If therapies exist, they tend to be very expensive - for obvious reasons. Given these problems the IOM was asked to review the situation and report its findings toward improving the R&D response toward therapies.

 The report calls for an integrated national strategy.  Let's call it 7 points of light:

• Public-private collaboration of government, commercial companies, academic institutions, and advocacy groups
• Application of science and technology that can make rare disease research and product development faster, easier and less expensive
• More trial design and use of analytical tools tailored to doing research in small populations
• Sharing research resources and infrastructure to better leverage scarce funding, data, biological specimens and rare disease-patient participation
• Reasonable rewards and incentives to private sector innovation and prudent use of public resources to meet targeted goals when appropriate
• Adequate staff with expertise on rare disease research and product development in public agencies funding biomedical research on rare diseases or regulating drugs
• Mechanisms for weighing priorities for rare disease research and measuring progress toward these goals

The IOM recommended the NIH develop a comprehensive action plan that covers all institutes and centers. The plan should cover program planning, grant review, training and coordination of all research. Further the NIH, should work with industry, academia, and voluntary organizations to develop a comprehensive system of shared resources for discovery research on rare diseases. This should include a public repository of available animal models (see Sigma-Aldrich SAGE Labs) for rare disorders that reflect disease mechanisms.  It should also include a public repository of biological and molecular mechanism data.

Mechanisms exist to encourage developement of orphan drugs for rare diseases like the Orphan Drug Act 1983 and NIH's Rare Diseases Clinical Research Network. IOM recommends more be done to identify and implement collaborative strategies with an eye toward reducing development costs. The NIH should coordinate the new Cures Acceleration Network with the Rare Diseases Clinical Research Network, the Clinical and Translational Science Awards program, and the grants programs of FDA's Office of Orphan Product Development.

Pulling it all together the IOM calls on the Secretary of DHHS to establish a national Task Force to see it through over the next 4 to 8 years.  Time to get busy.

 

Posted by Bruce Lehr October 7th 2010.

  

 

Pfizer Goes On Pipeline Diet

Pfizer has reduced the number of drugs in its pipeline according to a report in InPharm.   The project is of course necessitated by its acquisition of Wyeth as the company takes a hard look at which drugs will provide the greatest return - and help to replace revenue soon to be lost via patent expirations.

Pfizer says it will cut 31 programs overall.  Of the 31 programs to be cut, 15 are in phase I. Pfizer will still have  26 programs in phase III and 9 in registration.  It also has 27 products that are biologics and another 4 vaccines in development.

As announced earlier this year, Pfizer will place more emphasis on rare, orphan and neglected diseases - including new drugs for onchocerciasis (river blindness) and the fungal infection aspergillosis - often seen in immuno-compromised patients.

 

Posted by Bruce Lehr September 28th 2010.

Rich Markets Lure Biotech's from Developing World Cures

Fierce Biotech published the findings of a pair of Canadian researchers, Rahim Rezaie and Peter Singer, who studied 78 biotechs operating in India, Brazil, China and South Africa.  Many are working on affordable drugs for neglected diseases in the developing world.  But, will they continue to do so?

Ironically, making drugs for the developing world pushes them toward partnerships with companies with more marketing resources. These "superior" marketing organizations tend to be more focused on higher value drugs in Western markets that promise greater revenues. In many cases, this can cause the smaller biotechs to switch focus to these higher revenue applications.

Shantha Biotechnics has had a focus in low cost neglected areas.  It was responsible for developing a hepatitis B vaccine that is sold for $0.25 versus the $15 standard in the market.  The author's fear that Shantha could now switch its mission given its new owner - Sanofi-Aventis -- and trade global health for global wealth. 

Rezaie and Singer call on goverments and private sector entities to institute some/all of the following tactics to help support continued focus on neglected and low cost therapies by these emerging market biotechs:

• More public-private partnerships
• Advance market commitments to purchase neglected therapies
• Prizes to develop neglected drugs - for example a global health prize to recognize excellent examples of Southern [hemisphere] innovation against diseases of the poor
• Patent pools to encourage IP sharing to develop neglected drugs by lowering costs due to patent barriers, and to speed informatin exchange/sharing
• Priority review vouchers to encourage drug development and promote faster market entry with concomitant faster revenues
• Orphan drug-like legislation similar to that of US
• New financing sources like R&D for Affordable Healthcare (Wellcome Trust & India Dept of Biotechnology), PATH program (Seattle), a US SBIR-type program for small enterprises

"Taking steps now will ensure that innovative capacity is tapped not only for the industrialized countries but also in the emerging economies so that health needs of the poor can be more fully addressed."

Posted by Bruce Lehr September 16th 2010.

 

 

Would EU Vouchers Spur Neglected Disease Drug Development?

According to Professor David Ridley, Fuqua School of Business, Duke University and Alfonso Calles-Sanchez, patent expert in Spain's Patent Office, a "priority review voucher" award system in the EU would favor development of treatments for neglected diseases like tuberculosis, malaris, leishmanaiasis - as reported in MNT.

The voucher's would award a company expedited regulatory review through the EMA for one of its other drugs in return for developing a treatment for a neglected disease -- an innovative swapping concept.  Currenlty, these diseases effect more than 1B people but largely in poor and developing nations.  Historically, the financial incentive hasn't been strong enough to spur development of treatments.

For example, "If you develop a new drug for malaria, your profitable cholesterol-lowering drug could go on the market a year earlier," global health philanthropist Bill Gates explained at the 2008 World Economic Forum in Davos. "This priority review could be worth hundreds of millions of dollars," Gates said.

With expedited review, a company's drug not only reaches the market faster but preserves patent life for its drug in a commercial stage.  This is potentially worth hundreds of millions too. The proposed EU system would mirror a system already adopted in the US in 2007.  Novartis received the first priority review in the US in 2009, following development of its drug Coartem for malaria.

"A European priority review voucher system, combined with the U.S. system, would provide an extraordinarily powerful incentive for companies to invest in treatments for neglected diseases," Calles-Sánchez added.

Posted by Bruce Lehr September 11th 2001.

Biotech New Deals

A couple biotech deals on the books being reported this morning.

Firstly, Anchor Therapeutics has signed a deal valued at up to $480 M with J&J's Ortho group for access to its pepducin technology for the identifying G protein coupled receptor-targeted therapeutic compounds in oncology applications.  This includes Anchor's gpr39 metabolic therapy.  Anchor's technology appears to be in hot demand.  There are already GPCR drugs on the market that account for more than $40 B in annual sales.  Anchor's technology promised to acccess more of these targets and in a different way than samll molecules.  Earlier this year Anchor signed a pact with Novartis valued at up to $200 M.  The J&J deal calls for an undisclosed upfront payment, research funding, and milestones.  See Fierce Biotech and Xconomy stories.

 

Secondly, Acceleron signed a $498 M deal with Shire for its lead therapy, ACE-031.  Again, the deal is structured as a $45 M upfront payment with milestones that could reach as high as $453 M.  The deal would give Shire international marketing rights outside the USA. ACE-031 is in Phase IIb trials for Duchenne's Muscular Dystrophy.  It acts as an inhibitor with the activin receptor to stimulate muscle growth and build strength. There is no current approved therapy for the treatment of DMD so it is a very high unmet medical need. Shire see it as a good fit to add to its orphan drug portfolio. See Fierce Biotech and Xconomy.

 

Posted by Bruce Lehr September 9th 2010.

German Labs Advance Whole Genome Screening for Rare Disease

Over the past year, several market reports have predicted that in the new world of pharmaceuticals - post-patent cliff, post-blockbusters, post-biosimilar onset -- much more attention will be spent on developing drugs for orphan or rare disease applications.  As further evidence of this trend, several Big Pharma companies have in fact announced their investment into these areas. See examples from Pfizer and Sanofi.  Both the FDA and the NIH have discussed how they see their support increasing in this area.  And, the Senate passed a new bill that looks to spur funding and development in the rare disease arena.

With this backdrop, we have a report this morning, in Medical News Today, that molecular scientists at Max Planck and at Charite Universitatsmedizin Berlin have succeeded in a whole genome screening process to - in this case - identify disease genes for Mabry Syndrome in 3 patients. They were able to track down individual mutations in the genome and connect these with the disease.  The researchers relied on high throughput sequencing technology to identify the defect.

"It was like the proverbial search for a needle in a haystack.  We fished out solely the 22,000 genes from the entire genome, decoded their sequence and examined them for mutations.  Using new bioinformatic analyses, we were able to limit the number of mutation candidates to two, one of which is ultimately responsible for Mabry Syndrome" explains Michal Ruth Schweiger from Max Planck.

The methods developed here enable the identification of mutations even in very rare diseases and could help speed our introduction to the era of personalized medicine.  Certainly, these type of diagnostic tests will aid in drug discovery and development as well -- and will complement efforts by the other players described above.

Posted by Bruce Lehr September 7th 2010.

Pfizer Extends Rare Disease Profile with FoldRx Acquisition

Both Fierce Biotech and Xconomy have stories on Pfizer's latest acquisition to bolster its holdings in the orphan drug and rare disease area. FoldRx has a lead drug candidate, tafamadis, in Phase II for the treatment of TTR amyloid neuropathy. FoldRx also had just secured a fresh round of funding -- $29 M --in its bank account. FoldRx has already filed with European regulators for drug approval and is preparing its application with to the FDA.

This buy adds to Pfizer's plans to set up a rare disease center in Boston -- announced in June.  It also follows on the heals of a licensing deal in December 2009 with Protalix BioTherapeutics in Israle for a treatment for Gaucher's Disease. 

Said Pfizer's Geno Germano, division President, the FoldRx deal "will complement the current and planned future research and clinical development taking place in Pfizer's Specialty Care Neuroscience disease area."

 

 

Posted by Bruce Lehr September 1st 2010.

Senate Extends Carrot for Rare Pediatric Disease Drug Development

The Senate will begin pondering a new bill that will provide big cash incentives to pharmaceutical companies for taking on the development of drugs to combat rare pediatric diseases.  The bill would help drug companies gain quicker approval and first to market will get bigger shot at blockbuster revenues -- especially with shorter approval cycle.  At least that is the theory underpining the bill.

The bill gives priority review vouchers to firms pursuing rare diseases -- and is an extension of a bill originally passed for rare tropical diseases.  It also closes a loophole that allowed manufacturers to use vouchers with existing drugs (for new applications).  The bill is geared to "encourage" (force) developers to come up with new therapies.  See Fierce Biotech.

Posted by Bruce Lehr August 10th 2010.

 

NIH's Collins Clamors for Public War Chest to Fund Rare Disease Trials

Last week I wrote post about FDA playing bigger role in disseminating research results to spur progress with rare diseases.  Yesterday, Fierce Biotech published a story stating the Francis Collins of the NIH wants Congress to set aside a $500 M fund that would allow the NIH to back up to $15 M trials in biotechs pursuing rare disease indications.

This would represent a shift in emphasis from support of basic research toward more applied research - an innovation!  It also represents a possible new source of trial funding for the cash-strapped small biotechs who really lack as much access to capital as they once had. Collins goal is to secure funding for up to 20 new entitites.

 

Some in the more basic research camp are concerned that this shift in emphasis will rob Peter to pay Paul.  But, I think it makes good sense to try to capitalize on research that has already resulted in a discovery and attempt to make it more commercially viable.  One big gap now is sufficient funding for small biotechs to take their drugs through early clinical stages - a necessary step to secure either Big Pharma funding or a direct buy out from Big Pharma.  This could serve to help this along and is targeted to neglected disease areas so serves an additional good purpose to increase development in those areas known to be deficient now.

I hope the program secures funding so it can be tried.

Posted by Bruce Lehr July 9th 2010.

FDA Adopts Heightened Support of Orphan Drugs

Angie Drakulich, in PharmaTech Talk,  writes about new FDA policies to further increase its support of Orphan Drugs.

There are more than 6000 rare diseases - ones with patient populations below 200,000 in the US.  But, over 30 million Americans have one of those diseases. Since the inception of the Orphan Drug Act, 357 drugs and biologics have come to market.

Given these numbers, the FDA has increased its powers (or had its powers increased) to further help the situation.  Since 2007, the FDA has more authority to fast track an Orphan drug to approval.  It has been able to work with other regulatory agencies, like EMEA, to allow a single joint orphan-drug filing with both agencies. It's added a new Associate Director for Rare Diseases to its CDER branch that is in addition to its Office of Orphan Products Development (OOPD).

This month the FDA launched its new database to aid rare disease drug development. This was described recently on the FDA transparency blog and posted here. The database contains information on compounds that are already approved and could be applied to other diseases. As noted in the FDA's video, the agency believes it can play a major role in insuring Orphan Drug information is disseminated and shared.  That includes a training course to be held in October, and a two committee reports to Congress on rare diseases expected next year.

Frost & Sullivan earlier this year reported that rare and orphan diseases would be an increasing focus of the pharma industry in the next 5-10 years as a hedge against their flagging pipelines.  It is also reflective of the opportunity that personalized medicine may represent. The FDA seems to be plugging into the trend to help in any way it can which is good news for patients and their families.

Posted by Bruce Lehr July 2nd 2010.