Japan's Ministry of Labour, Health and Welfare (MLHW) recently
granted orphan drug status to
Aegerion Pharmaceuticals, Inc.
Juxtapid for the treatment of homozygous familial
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
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.
Alnylam Pharmaceuticals, Inc.
are looking to enter the hypercholesterolemia market.
Aegerion carries a Zacks #3 Rank (Hold). Currently,
looks better positioned with a Zacks Rank #1 (Strong Buy).
AEGERION PHARMA (AEGR): Free Stock Analysis
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