The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has put forward six new medicines for marketing authorisation, including two with orphan drug status.
Two orphan medicines - Biogen’s Alprolix (eftrenonacog alfa) and CSL Behring’s Idelvion (albutrepenonacog alfa) - have been endorsed for the prevention and treatment of bleeding in patients with haemophilia B.
Alprolix, a recombinant clotting factor therapy, is currently approved for the treatment of hemophilia B in the US, Canada, Japan, Australia and New Zealand, where it was the first haemophilia B therapy approved to provide prolonged protection from bleeds.
CSL Behring engineered Idelvion to extend the half-life of recombinant factor IX through fusion with recombinant albumin. The therapy is already cleared for use in Canada, and is under review in the US, Australia, Switzerland and Japan.
Servier’s Lonsurf (trifluridine/tipiracil, formerly TAS-102) was recommended for the treatment of metastatic colorectal cancer. The pill - combining an anticancer drug called trifluridine, able to interfere with DNA function, with tipiracil hydrochloride, which maintains blood concentration of the former - has shown a significant improvement in overall survival in patients taking Lonsurf compared to placebo, and a reduction in the risk of death of 32%
Elsewhere, Gilead Sciences’ Descovy (emtricitabine/tenofovir alafenamide, or TAF) received a positive opinion for the treatment of HIV-1 infection in adults and adolescents (ages 12 years and older with body weight at least 35 kg) in combination with other HIV antiretroviral agents.
TAF is a novel targeted prodrug of tenofovir that has shown high antiviral efficacy similar to and at a dose less than one-tenth that of Gilead’s Viread (tenofovir disoproxil fumarate, TDF). Because TAF enters cells more efficiently than TDF it can be given at a much lower dose, and the drug has also shown an improvement in renal and bone safety.
The CHMP also backed a marketing authorisation for Eli Lilly’s Taltz (ixekizumab) for the treatment of plaque psoriasis, on the back of data showing that for patients treated with the monoclonal antibody either every four weeks or every two weeks, between 78% and 90% of patients achieved at least a 75% reduction in the Psoriasis Area and Severity Index score at 12 weeks.
Hospira UK’s generic Palonosetron Hospira (palonosetron) picked up a positive recommendation from the Committee for the prevention of nausea and vomiting associated with chemotherapy.