Alexion Pharmaceuticals (ALXN) is looking to get its primary pipeline candidate, asfotase alfa, approved in Japan for treating patients suffering from hypophosphatasia (HPP), an ultra-rare genetic disorder. To that end, the biopharmaceutical company has filed a new drug application (NDA) with the Japanese Ministry of Health, Labour and Welfare (MHLW) for the candidate.
Alexion filed the application on the basis of encouraging data from multiple studies (n=71) on asfotase alfa in 71 HPP patients. The evaluated patients, including some from Japan, covered a wide spectrum in terms of age ranging from newborns to those aged 66 years. Data from a retrospective natural history study in infants and three pivotal prospective studies along with their extensions was considered in the filing.
The Japanese NDA will be reviewed on a priority basis, in case it is accepted by the MHLW. This is due to the fact that asfotase alfa enjoys orphan drug designation in Japan. This special status was awarded last month to the candidate by the MHLW (read more: Alexion's Asfotase Alfa Gets Japanese Orphan Drug Status). Alexion is also looking to get asfotase alfa approved in the U.S. and EU for HPP.
We expect Alexion to provide a detailed update on asfotase alfa’s regulatory status on its third-quarter conference call, scheduled for Oct 23. Approval of the candidate would reduce the company’s dependence on Soliris, currently the sole marketed product at Alexion.
Alexion currently carries a Zacks Rank #4 (Sell). Better-ranked stocks in the health care sector include Medivation (MDVN), Emergent BioSolutions (EBS) and Ligand Pharmaceuticals (LGND), each sporting a Zacks Rank #1 (Strong Buy).
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