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Orphan Drugs: Global Regulatory Events

January 27, 2014

This Blog Post discusses recent global non-US regulatory events for orphan drugs.

I – Europe

At a January meeting, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) gives the following opinions for three orphan drugs :

•   Positive recommendation for Bayer’s Adempas (Riociguat) for the treatment of Chronic Thromboembolic Pulmonary Hypertension (CTEPH) and Pulmonary Arterial Hypertension (PAH)

•   Negative opinion for AB Science’s Masiviera (Masitinib) which is intended for the treatment of non resectable locally advanced or metastatic pancreatic cancer

•   Negative opinion for PTC Therapeutics’ Translarna (Ataluren) which is intended for the treatment of Duchenne Muscular Dystrophy.

Also, EMA’s Committee for Orphan Medicinal Products (COMP) issues 15 positive opinions for ODD at their January meeting (Reference Blog Post). These ODDs are to be presented to the European Commission (EC) for final approval. If the EC approves these ODDs, the drugs receive ODD in the European Union (EU).

II – Mexico

Aegerion Pharmaceuticals’ orphan drug Juxtapid (Lomitapide) 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.

III – Japan

Takeda Pharmaceutical announces this month, that its New Drug Application (NDA) for Adcetris (Brentuximab Vedotin) is approved by the Japanese Ministry of Health, Labour and Welfare (MHLW), for the treatment of patients with CD30 positive relapsed/refractory Hodgkin Lymphoma (HL) or relapsed/refractory Anaplastic Large Cell Lymphoma (ALCL). The drug receives Orphan Drug Designation (ODD) from the MHLW in March 2012 for both HL and ALCL.

Also this month, the MHLW approves Bayer’s Adempas (Riociguat) to treat CTEPH. In September 2011, the MHLW grants ODD for this indication.

IV – Australia

Australia’s Therapeutic Goods Administration (TGA) grants on January 15, ODD for AstraZeneca’s Olaparib, as monotherapy for the maintenance treatment of adult patients with platinum-sensitive relapse BRCA-mutated ovarian, fallopian tube, or primary peritoneal cancer, who are in response to platinum-based chemotherapy.

Please Note: “Earth6391” courtesy of THEBLITZ1 at en.wikipedia [public domain].

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