Alexion’s late stage orphan drug granted “breakthrough drug” designation

In Regulation & Government by Tim Peplow

alexion orphan drug soliris alfa (

Alexion Pharmaceuticals, manufacturers of blockbuster orphan drug Soliris, has announced that the US Food and Drug Administration (FDA) has granted a Breakthrough Therapy designation to a separate late-stage orphan drug in Alexion’s pipeline (press release).

The drug, called asfotase alfa, is a first-in-class targeted enzyme replacement therapy for the treatment of patients with hypophosphatasia (HPP). HPP is an ultra-rare metabolic disorder that leads to progressive damage in multiple vital organs, and the FDA has confirmed that adult-onset HPP is “a serious and life threatening disease or condition”. A Breakthrough Therapy designation is part of the FDA Safety and Innovation Act (FDASIA) of 2012, and aims to expedite the development of a drug to treat a serious or life-threatening disease. Such a designation means that the FDA considers a drug to show substantial improvement over existing therapies for a serious and unmet medical need.

“The FDA’s Breakthrough Therapy designation for perinatal-, infantile- and juvenile-onset HPP recognizes the severe, debilitating and life-threatening nature of the disease, the clear unmet medical need of patients, and the clinical evidence collected to date on asfotase alfa,” said Martin Mackay, Ph.D., Executive Vice President, Global Head of R&D at Alexion, in a press release. “Asfotase alfa is a highly innovative therapeutic candidate with the potential to transform the lives of patients with HPP who currently have no treatment options and often receive only palliative care for this life-threatening disease.”

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Alexion’s Soliris is widely regarded as the world’s most expensive drug, and have already received approval in the US, EU and now Canada for ultra-rare atypical haemolytic uraemic syndrome (aHUS) and paroxysmal noctural haemoglobinuria (PNH). The orphan drug topped $1.1bn in sales in 2012. According to PMLive, analysts have suggested that asofase alfa could give Alexion a second big-selling product.

Do you think asfotase alfa will be another Soliris for Alexion? What do you think? Join our discussion on LinkedIn, or leave a comment below. Want more from Total Orphan Drugs? Sign up to our newsletter – it doesn’t cost anything and only takes a minute.

If you want to know more about innovation and strategy in the orphan drug market, you might be interested in attending the World Orphan Drug Congress Asia, 18-19 June 2013, Singapore, or the 4th World Orphan Drug Congress Europe, in Geneva on the 14th November 2013.

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