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Orphan Drugs: Global Regulatory Events (06.04.13)

June 4, 2013

Recent orphan drug global regulatory events are in the news. Here is a list and description of recent developments:

Aegerion Pharmaceuticals And Lojuxta (Lomitapide)

Aegerion Pharmaceuticals announces on May 31, 2013, that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP), gives a positive opinion with a unanimous vote recommending a marketing authorization for Lojuxta (Lomitapide) capsules (Juxtapid in the US), for Homozygous Familial Hypercholesterolemia (HoFH). The European Commission (EC) needs to ratify the CHMP positive opinion to grant marketing authorization in all 27 European Union countries. An approval decision is expected from the EC in the 3rd Quarter 2013.

The FDA approves of Aegerion Pharmaceuticals’ Juxtapid, the same Lomitapide drug for the same indication, in December 2012. Lomitapide has orphan drug status in both the US and the EU for HoFH.

There has been a competitive race in the US, between two marketed HoFH orphan drugs :

•   Aegerion Pharmaceuticals’ Juxtapid (Lomitapide), an oral once-a-day treatment
•   Genzyme/Isis Pharmaceutials’ Kynamro (Mipomersen).

Juxtapid’s price of $235,000-$295,000/year is much higher than Kynamro’s $176,000/year. Please reference a recent Blog Post for a detailed description of the two competitive drugs. Kynamro receives a negative opinion from EMA’s CHMP, in December 2012 and Genzyme/Isis Pharmaceuticals asks for a re-examination. In March 2013, Genzyme/Isis Pharmaceuticals announces that EMA’s CHMP confirms its previous position and maintains a negative opinion regarding the marketing authorization application for Kynamro.

Celgene And Pomalyst (Pomalidomide)

Celgene announces on May 31, 2013, that Pomalyst (Pomalidomide) receives a positive EMA CHMP opinion for the treatment of patients with relapsed and refractory Multiple Myeloma (rrMM). The indication is for the use of Pomalyst in combination with Dexamethasone for rrMM, in patients who have received at least two prior therapies, including both Lenalidomide and Bortezomib, and have demonstrated disease progression while on their last therapy. The EC usually follows the recommendation of EMA’s CHMP with an expected final decision within two to three months.

In February 2013, the FDA approves of Celgene’s Pomalyst Pomalidomide for patients with Multiple Myeloma (MM) whose disease progressed after being treated with other cancer drugs. Pomalyst is a pill that is meant for MM patients who have received at least 2 prior therapies, whose MM didn’t respond to these treatments, and the MM has progressed within 60 days of last treatment – Relapsed and Refractory MM (rrMM).

Pomalidomide has orphan drug status in both the US and the EU.

Please Note: “Globe” by Augiasstallputzer at Wikimedia Commons [Public domain] | Wikimedia Commons.

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

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