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 saabira.chaudhuri@dowjones.com
Subscribe to WSJ: http://online.wsj.com?mod=djnwires