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The New York Times reported today that French drug maker Sanofi Aventis has made a bid to purchase beleaguered orphan drug manufacturer Genzyme. According to the report, Sanofi approached Genzyme about two weeks ago about a possible sale. Sanofi is currently waiting for a response from Genzyme. If Genzyme rebuffs the takeover bid, persons close to the deal said that Sanofi may possibly try to acquire Genzyme via a hostile takeover bid.

Sanofi is facing revenue losses because many of its blockbuster products including the anti-clotting agents Plavix and Lovenox will or have lost patent protection. Plavix's patent expiry will occur in 2001 whereas Lovenox has already lost patent protection ( yesterday the FDA approved a generic version of the drug). Further, Sanofi, unlike most major pharmaceutical companies, is glaringly deficient in biotechnology products and has long been known to be seeking a quick entry into the biotech market. To that end, the Genzyme bid makes complete strategic sense to bolster sales and secure Sanofi's future.

Genzyme is the fifth largest biotechnology company in the world. Sales of it orphan drugs to treat Gaucher’s, Fabry and Pompe disease annually exceed $3.0 billion in sales even though they are used to treat small numbers of patients (<20,000).

Genzyme’s value has plummeted in the past year because of manufacturing problems and is currently operating under a US Food and Drug Administration consent decree after being fined $175 million by the agency. Many shareholders have called for the dismissal of Henri Termeer, Genzyme’s CEO for the past 25 years. To date, Termeer has refused to step down even though Genzyme’s stock continues to under perform. News of a possible takeover caused Genzyme’s stock price to soar; gaining more than 15 per cent on Friday to $62.50.

I believe that Sanofi is approaching Genzyme at the right time. Recently, Genzyme reached an agreement with Carl Icahn, who owns a substantially amount of stock, to prevent a proxy battle to reshape Genzyme’s board and oust Termeer. Also, another major shareholder, Ralph Whitworth, is unhappy with recent events at the company. Sanofi’s acquisition of Genzyme would provide a quick entry into the biotechnology and orphan drug markets and also appease shareholders like Icahn and Whitworth if the deal is rich enough. Also, Sanofi’s manufacturing experience would help Genzyme overcome its problems in that area.

Stay tuned for updates.

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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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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