Aegerion Pharmaceuticals: Juxtapid Sales Continue To Climb
Updated 01/20/14: With information on the approval of Juxtapid in Mexico for HoFH.
Aegerion Pharmaceuticals, a Cambridge, Massachusetts biopharmaceutical company focusing on development and commercialization of treatments for rare diseases, launches in the United States in January 2013, orphan drug Juxtapid (Lomitapide). Juxtapid is an oral once-a-day treatment for rare disease Homozygous Familial Hypercholesterolemia (HoFH). HoFH is caused by genetic defects inherited from both parents that affects the function of the LDL receptor, that is responsible for removing bad cholesterol (LDL-C) from the body.
Background Information On Juxtapid For HoFH
• Receives FDA Orphan Drug Designation (ODD) in October 2007
• Launches in US in January 2013
• US price of $235,000 – 295,000/year
• Boxed warning of potential for liver toxicity
• Restricted distribution through Risk Evaluation & Mitigation Strategies (REMS) requiring patient to have lab or clinical diagnosis consistent with HoFH.
Approval Of Juxtapid In Mexico
Aegerion Pharmaceuticals announces January 20th , that Juxtapid is approved by the Mexican Federal Commission for the Protection against Sanitary Risk (COFEPRIS) as an adjunct treatment to a low-fat diet and other lipid-lowering treatments, including LDL apheresis where available, 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 HoFH. In the approval of Juxtapid, COFEPRIS gave the medication recognition as an Orphan Drug.
J.P. Morgan Healthcare Conference
Marc Beer, CEO of Aegerion Pharmaceuticals, presents on January 13 at the J.P. Morgan Healthcare Conference, in San Francisco. Here are a few highlights from the presentation:
• 1 year ago at this same meeting, the company gives a figure of $15 – $25 million in revenue for 2013, with approximately 250 patients by the end of 2013
• For 2013, $48 – $49 million in Preliminary (Unaudited) Net Product Sales (reference graph in presentation):
- $ 1.2 million in Q1 2013
- $ 6.5 million in Q2 2013
- $16.3 million in Q3 2013
- Estimation of $25 million in Q4 2013
• For 2014, Revenue Guidance of $190 – $210 million in Net Product Sales
• 100% of Juxtapid prescriptions process from REMS certified physicians
• HoFH prevalence is underestimated; less than 1% currently diagnosed
• Will continue the growth of Juxtapid in the US for 2014, by more effective patient onboarding and support
• In November 2013, receives FDA Warning Letter about the interviews he gives CNBC’s Fast Money, and says that he should have chosen words that would have been more balanced in nature; working with FDA to resolve these concerns and plans on running a corrected broadcast in the near future on CNBC to correct any potential misimpressions and to reinforce the drug’s label and safety information
• Mentiones subpoena from the US Department of Justice for an investigation into the company’s sales & marketing practices; assures that all is within compliance regulations
• Comprehensive customer support for promoting access and adherence for maintaining HoFH patients on Juxtapid Therapy, through the company’s COMPASS Program, which provides:
- Customer care
- Reimbursement support
- Specialty Pharmacy services.
• European launch of Lojuxta (Lomitapide) on track & is approved in 27 European countries + Iceland & Norway
• Global Expansion with ongoing regulatory filings in key markets:
- Regulatory activity ongoing in > 45 countries
- Goal is to be in 85 countries on a global basis
- Currently have filed applications for market approval in Canada, Israel, Mexico, & Brazil
- ODD in Japan with plan to submit J-NDA following completion of 26-week efficacy phase
• Expansion into pediatric market:
- Non-clinical rodent study complete & analysis of data is in process
- Trial protocol discussions with both FDA & EMA underway
- Start of clinical study for 2014
- Potential pediatric US market: 1,000 – 1,500 patients.
FDA Warning Letter And A Subpoena
The positive preliminary sales results for 2013 are clouded by the receipt of :
1) A FDA Warning Letter, and
2) The subpoena from the US Department of Justice requesting documents regarding the marketing and sales of Juxtapid in the U.S.
Mr. Beer speaks to both of these topics in the presentation. According to an online January 17th article from fdanews.com :
”Aegerion Pharmaceuticals is under threat of a class-action shareholder lawsuit after government prosecutors earlier this month as asked the drug maker to hand over documents tied to sales and marketing ….. A demand for a jury trial has been filed in the US District Court for the District of Massachusetts by plaintiffs alleging the Cambridge, Mass.-based company misled investors and shareholders about its financial situation in the wake of an FDA warning letter …”.
Please Note: “#59180: Rock Climbing” from Federal Highway Administration, US Department of Transportation [Public Domain].