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Orphan drugs in latest CHMP recommendations

EMA advisers back new products from Genzyme, Boehringer and others
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Orphan drugs from Genzyme and Boehringer Ingelheim were among ten new medicines recommended for European approval by the EMA's scientific advisers.

The Committee for Medicinal Products for Human Use (CHMP) backed Genzyme's Cerdelga (eliglustat) and Boehringer Ingelheim's Ofev (nintedanib) in its latest round of opinions - which are usually followed by a full marketing authorisation within three months.

Cerdelga is an oral drug to treat the rare genetic condition type 1 Gaucher disease, which is defined by the lack of an enzyme to break down the fatty waste product glucosylceramide.

The condition leads to a build up of glucosylceramide in the body, including the liver, spleen and bone marrow, and can cause symptoms such as anaemia and enlarged organs.

The disease, which affects around 15,000 people in the EU, has limited current treatment options and the addition of oral Cerdelga would be a benefit for patients who tend to be treated with intravenous injections such as enzyme replacement treatment Cerezyme (imiglucerase), also made by Genzyme.

Cerdelga has demonstrated it is as effective as Cerezyme in clinical trials and was approved in the US in August.

The other orphan medicine recommended by the CHMP was Boehringer's Ofev, which received a positive opinion for the treatment of idiopathic pulmonary fibrosis (IPF), a progressive scarring of the lungs that has a median survival of two to three years after diagnosis.

The active ingredient of Ofev - nintedanib - is already recommended by the EMA as a lung cancer treatment but the drug has also demonstrated an ability to improve outcomes in IPF patients.

Two hepatitis C treatments from AbbVie

The CHMP's recommendations also saw AbbVie join the list of companies with a recommendation for a new hepatitis C drug.

In this case AbbVie received backing for two medicines: Exviera (dasabuvir) and the combination treatment Viekirax (ombitasvir, paritaprevir and ritonavir).

Both drugs were recommended for chronic hepatitis C infection, although they join a very competitive market that is currently led by Gilead Sciences' Sovaldi (sofosbuvir).

CHMP backs psoriasis drugs

The psoriasis therapy area also received a boost with recommendations for Cosentyx (secukinumab) from Novartis and Otezla (apremilast) from Celgene.

Cosentyx is an injectable treatment that has demonstrated superiority to Pfizer's psoriasis blockbuster Enbrel (etanercept) in clinical trials, while Otezla is on course to be the first oral PDE4 inhibitor approved in Europe for the treatment of patients with psoriasis and psoriatic arthritis.

Shionogi, MSD, Genzyme and Teva

Other drugs recommended by the CHMP included Shionogi's Senshio (ospemifene), which received a positive opinion for the treatment of vulvar and vaginal atrophy, and MSD's Zontivity (vorapaxar), which was recommended for the reduction of atherothrombotic events.

Completing the positive opinions were two new informed consent applications: Sevelamer carbonate Zentiva (sevelamer) from Genzyme for the control of hyperphosphataemia in adults receiving haemodialysis or peritoneal dialysis and Rasagiline ratiopharm (rasagiline) from Teva for the treatment of Parkinson's disease.

Article by
Thomas Meek

24th November 2014

From: Sales, Regulatory



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