Like Rodney Dangerfield, biotech companies achieving FDA product approval milestones are not getting much respect from investors these days. Take the case of Aegerion Pharmaceuticals Inc., of Cambridge, Mass.

On Dec. 24, the company won FDA approval for Juxtapid (lomitapide) capsules as an adjunct to a low-fat diet and other lipid-lowering treatments a few days ahead of its scheduled PDUFA date of Dec. 29. The product is targeted to reduce low-density lipoprotein cholesterol (LDL-C), total cholesterol (TC), apolipoprotein B (apo B) and non-high-density-lipoprotein cholesterol (non-HDL) in patients with homozygous familial hypercholesterolemia (HoFH).

The rare and serious genetic disease impairs the function of the receptor responsible for removing LDL-C ("bad" cholesterol) from the body. A loss of LDL receptor function results in extreme elevation of blood cholesterol levels. HoFH patients often develop premature and progressive atherosclerosis, a narrowing or blocking of the arteries.

Despite the approval of its first product, the company's share price (NASDAQ:AEGR) closed Monday at $25.25, down 46 cents, in heavy volume albeit in a holiday-shortened trading day with the markets closing early. When markets closed on Wednesday, the stock had edged up to $2.73, a gain of 48 cents.

Just a couple of months ago, lomitapide, a microsomal triglyceride transfer protein inhibitor, gained a 13-2 nod for approval from the FDA's Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC). (See BioWorld Today, Oct. 16, 2012, and Oct. 18, 2012.)

The FDA based its approval of Juxtapid on Aegerion's pivotal Phase III study, which evaluated the safety and effectiveness of the medicine to reduce LDL-C levels in 29 adult patients with HoFH.

In a conference call, Marc Beer, CEO at Aegerion, said the label for Juxtapid will contain a boxed warning for the risk of hepatotoxicity. The REMS will require the certification of health care providers/pharmacies, and the company will also establish a voluntary registry of 300 patients worldwide who will be monitored for a period of 10 years to better characterize the drug's long-term effectiveness.

Launch of Juxtapid is planned for January, and more details on the launch and price of the product could come during the company's presentation at the upcoming JP Morgan Healthcare conference in San Francisco. Beer did say that their trained sales force is ready to go and that product pricing will be in the range of $200,000-$300,000 per patient, per year.

An estimate of patient population in the U.S. is about 3,000. Beer also said that they were predicting a European regulatory decision on the product around mid-2013.

The FDA was very busy in the run-up to the holidays. Late Friday, Dec. 21, it gave the green light to Mountain View, Calif.-based Alexza Pharmaceuticals Inc. for Adasuve (Staccato loxapine) inhalation powder 10 mg for the acute treatment of agitation associated with schizophrenia or bipolar I disorder in adults.

However, Alexza's shares suffered the same fate as those of Aegerion tumbling 80 cents (13.8 percent) in very heavy trading to close Monday at $4.99.

In May the company received a complete response letter to its new drug application for Adasuve citing manufacturing deficiencies. (See BioWorld Today, May 7, 2012, and Dec. 26, 2012.)

Investors made it three for three Monday with shares of NPS Pharmaceuticals Inc. falling 15 cents. Earlier on Friday the company received FDA approval for Gattex (teduglutide) for short bowel syndrome (SBS) more than a week ahead of its Dec. 30 scheduled PDUFA date. (See BioWorld Today, Dec. 26, 2012.)