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The recent manufacturing woes of orphan drug manufacturer Genzyme have been well documented and widely publicized. These problems resulted in massive shortages of some of its top selling drugs Cerezyme (Gaucher disease) and Fabrazyme (Fabray disease) causing many of the patients who depend on these drugs to maintain their quality of life to go without reduced or no treatments for months. 

Because both drugs were approved by the US Food and Drug Administration (FDA) as orphan drugs they enjoy seven years of market exclusivity from the date of regulatory approval which prohibits other companies from seeking approval and selling similar drugs in the US. Consequently, Genzyme is the only commercially-available source for the drugs. Genzyme’s ongoing biomanufacturing created massive shortages of both drugs last summer. Because of this, FDA allowed two other companies, Shire and Protolix Biotherapeutics (both have treatments for Gaucher (Protalix) and Fabray (Shire) disease in late stage clinical development), to make their drugs available (at no charge) to patients prior to regulatory approval. This was a relatively rare and bold move by the agency. But, to be fair, they had little choice because so many patients were suffering.  

In case you may be wondering there are approximately 1500 Cerezyme users and fewer than 1000 Fabrazyme users in the US. Despite the small numbers of patients, the cost of the treatments are extraordinarily high; costing patients as much as $200,000 per year for treatment.

According to an article in today’s NY Times as many a 15 percent of American Cerezyme users have switched to a different drug. Fewer patients with Fabray disease have switched mostly because Shire’s drug for Fabray disease Replagal was less widely available. The almost year-long shortages of both drugs have seriously tarnished Genzyme’s reputation. And previously loyal patients are questioning their almost decade long allegiance to the company. This, coupled with a 26 per cent decline in Genzyme’s stock price last year suggest that Genzyme’s standing as one of the top five biotechnology companies in the world may be in serious jeopardy. 

Despite calls for his resignation (and an attempt by Carl Icahn to wrest control of the company), CEO Henri A. Termeer, who has led Genzyme for over 20 years has vowed not to resign. While manufacturing problems are not uncommon in the biotechnology industry, the severity and ongoing nature of the manufacturing problems at Genzyme’s Allston Landing, MA production facility are unacceptable; especially for a company that specializes and prides itself in developing treatment for orphan disease indications. The FDA recently announced that it would fine Genzyme and place its manufacturing operations under a consent degree for an indefinite period of time.

Genzyme representatives now contend full supplies of Cerezyme will be available after May 1 and those for Fabrazyme possible in the third quarter of this year.

While so-called “copycat” or “me too” drugs developed by pharmaceutical companies tend to be vilified by consumers and patient advocacy groups, the agency prefers to approve more than one treatment option for a given disease indication in case one medication doesn’t deliver the intended therapeutic benefits or induces untoward adverse events. Unfortunately, the seven years of market exclusivity awarded to orphan drugs manufacturers that garner regulatory approval for their products prohibits this. The biomanufacturing fiasco at Genzyme suggests that it may be time to reexamine the Orphan Drug Act and modify some of the financial and regulatory terms that were included to induce drug companies to develop new treatments for orphan disease indications.

Until next time…

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

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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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Genzyme announced in a Securities and Exchange Commission filing on Monday that it inked a "fill and finish manufacturing services" deal with Hospira for several of its top selling drugs including Cerezyme (Gaucher disease), Fabrazyme (Fabry disease, Myozyme (Pompe disease) and Thyrogen (thryroid cancer). The move follows a series of highly publicized manufacturing problems at the company’s Allston Landing, MA biomanufacturing facility in 2009.

Beginning in March, Genzyme received a warning letter from the US Food and Drug Administration (FDA) detailing "significant objectionable conditions" at the Allston facility. The agency identified deviation and violations of current Good Manufacturing Practice (GMP) in four areas including: 1) maintenance of equipment, 2) computerized systems, 3) production controls and 4) the failure to follow procedures aimed at preventing microbiological contamination.

In June, Genzyme shut down the biomanufacturing plant to clean up viral contamination that had been slowing down production of Cerezyme and Fabrazyme. The virus, Vesivirus 2117, is known to interfere with the growth of Chinese hamster ovary (CHO) cells and is believed to have been introduced through a cell culture nutrient. The virus doesn’t infect humans, but the shutdown cost the company millions in revenue and caused shortages of Cerezyme and Fabrazyme. Production restarted at the plant in September.

Meanwhile, in November, the Food and Drug Administration said it found tiny particles of steel, rubber and fiber in finished vials of Cerezyme, Fabrazyme, Myozyme, Aldurazyme (mucopolysaccharidosis I) and Thyrogen. These and other violations are outlined in a 483 that was issued by FDA following inspection of the troubled facility.

The deal with Hospira, which makes drug and medication delivery systems,calls for the initial term to expire on Dec. 31, 2015. There are options for a two-year extension. The financial terms of the agreement were not disclosed. The deal is still subject to regulatory approval for manufacturing the products.

While GMP deviations and warning letters are common place at many biotechnology companies, Genzyme’s ongoing manufacturing problems had potentially grave medical implications. This is because, unlike most of its competitors, Genzyme focuses almost exclusively on the development of orphan drugs. Orphan drugs are used to treat diseases like Gaucher, Fabry and Pompe disease which are rare, afflict relatively small numbers of patient and usually result from genetic mutations. Generally speaking, there is usually only a single manufacturer of orphan drugs. Consequently, manufacturing problems can result in drug shortages which may inhibit access to these life saving drugs. As corny as it may sound, patients with orphan diseases have literally placed their lives in the hands of the drug companies that manufacture these orphan drugs.

Until last year, Genzyme had an outstanding and impeccable reputation and was regularly lauded by the orphan drug community. Unfortunately, its management team lost sight of its original to commitment to quality—a sign that changes may be necessary in the executive suite. Hopefully, the new fill and finish deal with Hospira will eliminate many of the company’s manufacturing problems and Genzyme can restore confidence in its brand!

Until next time....

Good Luck and Good Manufacturing !!!

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