The U.S. Food and Drug Administration announced it had approved an Aegerion Pharmaceutical drug for a cholesterol disease that is very rare, but the drug will require safety precautions due to its potential liver toxicity.
Juxtapid, the approved drug, is the first drug approved for Aegerion. Now investors will watch to see if the company will be able to navigate the commercializing challenges for the newly approved drug for the rare disease.
Juxtapid will have a warning label that is prominent and other requirements will be needed that could cause a four month delay before new patients can receive the treatment.
Marc Beer, the CEO of Aegerion, said the requirements consistent with those that the company had proposed to the regulatory agency. The warnings will ensure the patient’s safety and long term adoption to the product. Beer said he believed the FDA was right about the drug and understood the disease.
The drug will be utilized to help patients who have homozygous familial hypercholesterolemia or HoFH. This is a rare genetic disorder causing potentially fatal buildups of bad cholesterol known as LDL-C.
Estimates are that more than 3,000 patients in the U.S. are eligible to start using the drug. The cost annually for the therapy is thought to be up to $300,000. Aegerion will have to train and then certify physicians, pharmacies and patients, while insurers must have payments pre-authorized for reimbursement prior to the drug being distributed to patients. The entire process could take up to four months before the patient actually has the medication to take, said Aegerion officials.
In January, the sales process will start for Juxtapid. Aegerion said more details would be provided about the launch of the drug, the sales force and financial guidance for the company for 2013. The company also said it was expecting a decision by regulators in the European Union sometime in mid-2013.
On Monday, shares of Aegerion stock fell to $25.25.