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A rare or orphan disease is defined in the US as one that affects fewer than 200,000 at any given time. It is estimated that there are 6000 to 8000 rare diseases in the world today. Because the number of patients afflicted with orphan diseases is so small, drug companies have historically been reluctant to invest money to discover and develop new treatments for them. The dearth of treatments for rare diseases induced Congress to pass the Orphan Drug Act in 1983 which provided market exclusivity, tax breaks and incentives and regulatory help for companies to development new drugs for orphan disease indications.

While many current blockbuster drugs including recombinant human insulin, growth hormone and erythropoietin originally garnered regulatory approval after receiving orphan status in the late 1980s, most big pharma and biotechnology companies (except Genzyme) largely abandoned orphan drug development until recently. The renewed interest in orphan drug development has been primarily driven by the demise of big pharma’s blockbuster business model that began in the early 2000s. The search for new, non-blockbuster drugs and fresh markets is what induced Pfizer, the world’s largest pharmaceutical company, to recently inked a multimillion dollar deal with Protalix Biotherapeutics, a small biopharmaceutical company developing a new treatment for Gaucher disease—an orphan indication.

Because of renewed interest and the ever increasing need for new orphan drugs, the FDA’s Office of Orphan Products Development is offering an Orphan Drug Designation Workshop that will provide a unique opportunity for all potential drug sponsors—including biotechnology companies, pharmaceutical firms and academic institutions—to learn about the application process for orphan drug designation.

The National Organization for Rare Disorders (NORD) is a co-sponsor of the workshops, which will take place on February 25-26 at Keck Graduate Institute and August 3-4 at the University of Minnesota.

Participants are encouraged to bring specific product proposals for at least one candidate orphan drug that holds promise for the treatment of a rare disease. A significant portion of the workshop will be dedicated to preparing applications, including one-on-one guidance sessions with FDA staff members. FDA will keep product and disease information confidential.

Final applications can be submitted to the FDA at the close of each workshop. For information or to register:

FDA Workshop Brochure
Registration for the February Workshop

Finally, February 28^th is Rare Disease Day. The event is sponsored by the EURODIS a European advocacy group that promotes awareness and research for rare diseases. NORD and Discovery Health are also sponsoring the day.

Until next time....

Good Luck and Good Job Hunting!!!!!!!!!

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Harrison Ford’s new movie “Extraordinary Measures” (also starring Brendan Fraser) is loosely based on John Crowley’s ongoing crusade to find a cure for Pompe Disease a genetically inherited illness that afflicts two of his three children.The film chronicles the 'extraordinary measures' taken by Crowley to find a treatment for the so-called orphan disease that affects the lives of about 40,000 persons worldwide. While I haven’t seen the film, it bears a striking resemble to the 1992 film “Lorenzo’s Oil” which chronicled the struggles of two parents to find a “cure” for their son’s adrenoleukodystrophy an another orphan disease.

Crowley’s story began about 12 years ago when his oldest child was diagnosed with Pompe Disease. For those of you who may not know, Pompe Disease is a progressive, multisystemic, debilitating, and often fatal neuromuscular disorder. The disease is linked to an inherited deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA), which is responsible for the breakdown of glycogen inside the cells. The result is intralysosomal accumulation of glycogen, primarily in muscle cells, that leads to a progressive loss of muscle function and ultimately death. At the time of the diagnosis, Crowley, a Princeton, NJ resident, was working as a marketer for Bristol Myers Squibb. He quickly learned that there was no effective treatment for Pompe Disease and that his daughter may not live beyond early childhood. Further, because the disease afflicted so few individuals, no pharmaceutical or biotechnology companies were working on treatments for Pompe Disease. 

To stave off the likelihood of his daughter’s death, in 2000, Crowley raided his 401k plan and mortgaged his home to start a company called Novazyme that focused exclusively on developing treatments for Pompe Disease. Having no time to waste, Crowley and the Novazyme team worked feverishly to develop an alglucosidase alfa enzyme replacement therapy for Pompe. By 2001, the Novazyme team had identified a likely treatment and Crowley sold his company to Genzyme. As a senior vice president at Genzyme, he oversaw clinical development of the product which is now called Myozyme and is the first FDA-approved treatment for Pompe Disease. Crowley left Genzyme in 2004 and is currently CEO of Amicus Therapeutics a 100 person company focused on developing new treatments for Pompe Disease and other orphan indications.

At present, there are no other treatments besides Myozyme for Pompe Disease. This is because Pompe Disease is designated as an orphan indication and Genzyme received seven years of market exclusivity for Myozyme as stipulated in the Orphan Drug Act. Myozyme received FDA approval in 2006.

While Genzyme has been the only player in the Pompe Disease market for the past four years, manufacturing and scale up problems threaten to jeopardize the Myozyme franchise. Genzyme’s highly publicized problems at its Allston, MA-manufacturing facility have been well documented and Genzyme’s management team is taking bold steps to correct them (including hiring a new senior vice president for global product quality) and entering into an agreement with Hospira Worldwide Inc to provide fill and finish manufacturing services.

But perhaps more troubling, were the problems that the company experienced when attempting to scale up Myozyme production from the 160L to 200L bioreactor scale to meet growing demand for the drug.  FDA informed Genzyme that that Myozyme® (alglucosidase alfa) produced at the 160L bioreactor scale and Myozyme produced at the 2000L scale should be classified as two different products because of differences in the carbohydrate structures of the molecules. And, the company would have to file a new biologics application (BLA) for the 2000L product to garner regulatory approval.

Currently, Genzyme has U.S. approval to sell Myozyme manufactured at the 160L scale, and the company has been seeking clearance from the FDA for Myozyme produced at the 2000L scale (now marketed as Lumizyme). Lumizyme has already been approved in more than 40 countries. However, manufacturing problems and violations at the Allston facility forced FDA to delay a decision on the approvability of Lumizyme this past March. Earlier this week, Genzyme announced that FDA will issue a new decision on Lumizyme in June.

While originally spurned by large drug companies, orphan drug development is becoming much more attractive because of the lack of new blockbuster drugs in most company’s development pipeline. According to a recent report, the number of orphan product designations in the US more than doubled in the last decade rising from 208 in the 2000-02 periods to 425 in 2006-08. More recently, Pfizer, the world’s largest pharmaceutical company announced that it agreed to pay at least $60 million for rights to Protalix Biotherapeutics Inc.'s new treatment (taliglucerase alfa) for Gaucher’s Disease another orphan indication. This suggests that Pfizer has made a decision to directly compete with Genzyme, the world leader in orphan drug development.

Don’t be surprised when other large pharmaceutical and biotechnology companies announce plans to compete in the orphan drug market...there is money to be made!

Until next time...

Good Luck and Good Job Hunting!!!

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