The FDA has approved Aegerion's ($AEGR) new drug for homozygous familial hypercholesterolemia, clearing a path to begin commercialization as the biotech comes to a major turning point. Lomitapide will now hit the market as Juxtapid featuring a boxed warning about the distinct threat of liver toxicity.
"We are excited that Juxtapid will become a new treatment option for patients with HoFH," said Marc Beer, the CEO at Aegerion. "The approval of our first product also marks an important corporate milestone for Aegerion and reflects our commitment to help patients in need."
Homozygous familial hypercholesterolemia is an extremely rare genetic condition characterized by dangerously high levels of LDL which can trigger a lethal set of cardiovascular conditions. A significant percentage of patients in the small pivotal study Aegerion mounted demonstrated a spike in liver enzymes, a classic red flag for drug developers. A REMS program will be required to monitor patients and educate physicians.
The approval marks a big milestone for the biotech, which will handle commercialization. Aegerion has been growing a small commercial operation, bringing its total staff count to about 70. That's a big change over just a couple of years. And the biotech will keep its eyes focused on lomitapide, with no other programs competing for attention. CEO Marc Beer told FierceBiotech recently that an OK would put the company on a path to profitability in 2014, completing a turnaround he started two years ago.
Lomitapide came up for FDA review at about the same time as mipomersen, from Isis ($ISIS) and Sanofi ($SNY). But that HoFH drug faces a much more problematic future. An EMA committee recently voted against approval, raising red flags over some severe risks associated with the treatment. And only a slight majority of FDA experts gave the treatment a thumbs-up, signaling that it may face tougher odds on a formal approval.
- here's the press release
- see the story from Bloomberg