Pfizer continues on rare disease path with new R&D unit

By Wai Lang Chu

- Last updated on GMT

Pfizer continues its foray into rare diseases as it creates a new R&D unit to investigate novel therapeutics for conditions such as Haemophilia. The pharma giant joins GlaxoSmithKline and Novartis as companies looking into producing specialised drugs as a way to offset slowing sales growth and loss of blockbuster patent protection.

The new unit created within Pfizer’s Worldwide Research and Development operation aims to capitalise on Pfizer’s existing research into such conditions as Haemophilia. In the past, Pfizer have also expressed its interest in treatments for muscular dystrophy and other conditions caused by genetic mutations.

The creation of an R&D unit represents a significant strategy shift into rare diseases for the firm. In December of last year, Pfizer agreed to pay $60 million initially with a further $55 million to license the worldwide rights to Protalix Biotherapeutic's drug for Gaucher disease.

This shift also produced job cuts as the company announced last month its intention to shed roughly 6,000 manufacturing jobs worldwide over the next five years.

This includes approximately 300 at the Andover biotechnology plant Pfizer took over when it bought Wyeth Pharmaceuticals for $68 billion last year.

With the impending expiration of blockbusters and a slowdown in emerging pipeline candidates, industry insiders believe pharma’s passing interest in specialised markets is gaining increasing momentum.

Drugs produced for ultra-orphan diseases with high unmet needs have an easier time commanding high prices. Genzyme’s Cerezyme, another drug used to treat Gaucher disease can command prices of up to $200,000 a year while medicines for cancer can cost $50,000 or more a year.

In addition, the growing trend of pharma companies using biomarkers to improve on current methods to define populations of patients increases the prospect of smaller markets suddenly becomes more attractive and financially viable.

Pfizer joins a growing band of companies who are currently pursuing treatments for rare diseases. In March, GlaxoSmithKline (GSK) steeped up its efforts to build a rare diseases business which saw the UK pharmaceutical major launch its own dedicated R&D unit.

April also saw GSK partner up with US firm Isis Pharmaceuticals in a deal that uses Isis’ antisense discovery platform to find and develop six drugs for rare illnesses including infectious diseases and some conditions causing blindness.

The deal is the second firm the UK giant has teamed up with. Dutch biopharma group Prosensa became the first with an agreement, signed last October that focused on the development of RNA drugs for the treatment of Duchenne Muscular Dystrophy (DMD).

In June, the Food and Drug Administration (FDA) granted approval to Novartis to allow the drug Ilaris to be sold as a therapeutic for cryopyrin-associated periodic syndrome. Only 300 Americans suffer from this syndrome, an inflammatory condition caused by a gene mutation.

David Simmons, president of Pfizer’s established products business unit, said in an interview with the New York Times​: “This is the first step in the pursuit of a formal strategy around orphan drugs and rare diseases.”

While there might low numbers of patients with these diseases, he said, “collectively, it’s a very large patient population with a great unmet medical need.”

Pfizer chief executive Jeff Kindler similarly commented on the reasoning behind his company’s change of direction stating the deal with the Israeli-based Protalix was, “the first of many. We’re trying to meet unmet medical needs in a way we haven’t done before.”

On the day the Protalix deal was disclosed Kindler commented: “Diseases that otherwise might not be well served are an opportunity for us.”

Of the more than 6000 diseases classified as orphan diseases, fewer than ten percent have therapies that directly address the underlying disease. According to the National Organization for Rare Disorders (NORD), a disease is considered rare in the U.S. if it affects fewer than 200,000 people. There are nearly 7,000 such diseases affecting nearly 30 million U.S citizens.

“Approximately 30 million Americans, 30 million Europeans and millions more around the world have rare diseases and for those people, there is no specific treatment,”​ said Peter L. Saltonstall, President and CEO of the National Organisation for Rare Disorders (NORD).

“We are hopeful that this research unit will lead to additional new medicines for patients suffering from devastating illnesses for which there is no cure,”​said Jose Carlos Gutierrez-Ramos, senior vice president, BioTherapeutics Research and Development.

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