The European Medicines Agency is recommending approval of a new medicine for haemophilia A developed by Roche that is intended to prevent bleeding or reduce the frequency of bleeding episodes in patients who have developed resistance to existing medicines.
A cancer drug that was dropped from development in 2011 but put back into clinical testing two years later with a different group of patients, has received approval for a third indication from the US Food and Drug Administration. The newest approval is to treat patients with certain types of breast cancer whose tumours have a BRCA gene mutation.
The first gene therapy for patients with an inherited retinal disease was authorised by the US Food and Drug Administration on 19 December, bringing the number of approved gene therapies in the US up to three. Luxturna (voretigene neparvovec-rzyl), was developed by Spark Therapeutics Inc.
The European Medicines Agency has issued a positive opinion for an allogeneic stem cell therapy to treat complex perianal fistulas in patients with Crohn’s disease. The product, Alofisel (darvadstrocel), was developed by TiGenix NV of Belgium.
The Italian reimbursement authority has reached an agreement with MolMed SpA under which it will reimburse the company’s cell therapy for patients who have received a stem cell transplant at €149,000 per infusion. Treatment can require up to four infusions.
The US Food and Drug Administration has approved a follow-on, or biosimilar, medicine for diabetes from Sanofi SA that is intended to improve the control of blood sugar in children and adults with both Type 1 and Type 2 forms of the disease.
Amsterdam is to be the new home for the European Medicines Agency following Britain’s decision to leave the European Union, and give up its right to host EU institutions at the end of March 2019. The decision was announced on 20 November by the EU General Affairs Council.
The US Food and Drug Administrations has issued four guidance documents explaining how it intends to regulate products derived from human cells and tissues. The guidance updates rules that first took effect in 2005 and includes new incentives for developers of potentially life-saving therapies.
The European Medicines Agency has recommended a new medicine that can prevent cytomegalovirus (CMV) reactivation and disease in patients who have received an allogeneic stem cell transplant. The medicine, Prevymis (letermovir), was developed by Merck & Co Inc.
A new monoclonal antibody treatment for multiple sclerosis (MS) has been recommended for approval by the European Medicines Agency following clinical studies that showed significant benefits for patients with both relapsing and primary progressive forms of the disease.