Japan's Ministry of Labour, Health and Welfare (MLHW) recently granted orphan drug status to Aegerion Pharmaceuticals, Inc.’s Juxtapid for the treatment of homozygous familial hypercholesterolemia (HoFH).
As per the requirements of Japanese regulatory authorities, Aegerion is conducting a pharmacokinetic/pharmacodynamic (PK/PD) study on Juxtapid, which will be over in the final quarter of 2013. Thereafter, Aegerion will conduct a small therapeutic study with Juxtapid in adult HoFH Japanese patients. Aegerion expects to file a New Drug Application (NDA) in the first half of 2015 in Japan.
We note that, in late Jul 2013, the Pharmaceuticals and Medical Devices Agency (PMDA) had recommended orphan drug status for Juxtapid in Japan.
Juxtapid was launched in the U.S. in late Jan 2013 for the treatment of patients suffering from HoFH. Juxtapid gained EU approval under the trade name Lojuxta in Aug 2013.
We are encouraged by Juxtapid’s performance. Net product sales for the second quarter were $6.5 million. At the end of Jul 2013, there were 215 patients under Juxtapid therapy as compared to 75 patients at the end of Apr 2013. As per estimates provided by Aegerion, there are at least 3,000 potential patients for Juxtapid in the U.S. We expect investor focus to remain on the sales ramp up of the drug.
Currently, companies like Regeneron Pharmaceuticals, Inc. (REGN - Free Report) and Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) are looking to enter the hypercholesterolemia market.
Aegerion carries a Zacks #3 Rank (Hold). Currently, Actelion Ltd. looks better positioned with a Zacks Rank #1 (Strong Buy).