Gentium Gets Negative Opinion From CHMP; Biogen, Others Get Positive Opinions

   By Saabira Chaudhuri 
 

Gentium SpA (GENT) said the European Medicines Agency's Committee for Medicinal Products for Human Use issued a negative opinion, recommending the refusal of marketing authorization for its treatment for hepatic veno-occlusive disease.

Gentium said the decision pertains to its Defibrotide drug to treat and prevent hepatic veno-occlusive disease in adults and children undergoing hematopoietic stem cell transplantation therapy.

The drug company said it remains convinced of the favorable benefit/risk profile of Defibrotide, noting this is the only option recommended by the European Group for Blood and Marrow Transplantation for the treatment veno-occlusive disease, an unmet medical need where no agents are currently approved.

Gentium will appeal the EMA's decision and request a re-examination of the CHMP opinion.

Gentium was halted premarket in New York and has declined 28% so far this year.

Separately on Friday, Biogen Idec Inc. (BIIB), Baxter International Inc. (BAX), Halozyme Therapeutics Inc. (HALO), Gilead Sciences Inc. (GILD) and Sanofi's (SNY) Genzyme unit all said they had received positive opinions from the CHMP.

Biogen said the positive opinion recommends marketing authorization be granted for Tecfidera as a first-line oral treatment for adults with relapsing-remitting multiple sclerosis.

The CHMP's recommendation is now referred to the European Commission, which grants marketing authorization for medicines in the European Union. It added the U.S. Food and Drug Administration is expected to make a decision on the marketing application for the drug in the U.S. in the coming days.

Baxter and Halozyme Therapeutics said the CHMP has granted a positive opinion for the use of HyQvia solution for subcutaneous use as replacement therapy for adult patients with primary and secondary immunodeficiencies.

Meanwhile, Gilead said the CHMP has adopted a positive opinion on its application for the once-daily, single-tablet regimen Stribild for the treatment of HIV-1 infection in certain adult patients.

Genzyme also announced it had received a positive opinion from the CHMP regarding the approval of once-daily, oral Aubagio for the treatment of adult patients with relapsing-remitting multiple sclerosis.

However, the CHMP didn't recommend that Aubagio receive a new active substance designation.

"We are very disappointed about the CHMP opinion regarding new active substance designation," Chief Executive David Meeker said. "We are considering all options and planning to request a re-examination of the new active substance designation."

Write to Saabira Chaudhuri at [e-mail]

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