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Aegerion Pharmaceuticals, Orphan Drug Juxtapid (Lomitapide), And The J.P. Morgan Healthcare Conference

January 9, 2013

       On Tuesday, January 8, 2013,3:00 PM PST/5:00 PM EST, Aegerion Pharmaceuticals gives a presentation at the J.P. Morgan Healthcare Conference in San Francisco. The presentation is given by Aegerion Pharmaceuticals’ CEO, Marc Beer.

Here are a few highlights from the presentation on the launch of orphan drug Juxtapid (Lomitapide) for the treatment of Homozygous Familial Hypercholesterolemia (HoFH):

1) FDA approval December 21, 2012, with boxed warning for potential for liver toxicity

2) Restricted distribution through REMS (Risk Evaluation & Mitigation Strategy) :

– Requirement that patient has lab or clinical diagnosis consistent with HoFH

– Education of prescribers about hepatotoxicity & need to monitor patients

3) Once-a-day capsule

4) Rare disease sales team with 100% predominant orphan drug sales experience

5) Approximately 1,800 US practitioners who treat 70 – 80% of HoFH patients :

– 100 academic lipid specialists

– 400 community lipid specialists

– 1,300 target community clinical cardiologists.

6) Identification of 1,500+ HoFH patients globally

7) By end of 2013, 250 – 300 patients globally on therapy & $15 – $25 million net revenue

8) Has met with 80% of the largest commercial payers

9) Establishment of the COMPASS Program to provide patient & physician support

10) Potential patient population target : 3,000 US & 3,000 in 5 EU countries

11) US gross price/patient: $235,000 initiation & $295,000 maintenance

12) European Medicines Agency (EMA) decision expected Mid-2013.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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