Seven new medicines, including one advanced therapy, recommended for approval

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended seven new medicines, including one advanced therapy medicinal product (ATMP), for approval at its March meeting.

The CHMP recommendedgranting a marketing authorisation for Strimvelis, a new gene therapy intended for the treatment of patients with adenosine-deaminase-deficient severe combined immunodeficiency (ADA-SCID), who have no matching donor for a stem cell transplant. Strimvelis has an orphan designation and is an ATMP.

The Committee recommended granting a conditional marketing authorisation for Darzalex (daratumumab) for the treatment of relapsed and refractory multiple myeloma. Darzalex has an orphan designation and was reviewed under EMA’saccelerated assessment scheme.

The Committee recommended granting a marketing authorisation for Galafold (migalastat) for the treatment of Fabry disease, a rare genetic disorder. Galafold has an orphan designation. For more information, please see the press release in the grid below.

Pandemic influenza vaccine H5N1 MedImmune also received a positive opinion from the CHMP. This is the first pandemic live attenuated influenza vaccine against avian influenza (H5N1) to be recommended for approval in the European Union (EU). The vaccine is intended for pandemic preparedness.

A biosimilarmonoclonal antibody, Flixabi (infliximab), was granted a positive opinion by the Committee for the treatment of rheumatoid arthritis, adult and paediatric Crohn’s disease, ulcerative colitis, paediatric ulcerative colitis, ankylosing spondylitis, psoriatic arthritis and psoriasis.

The CHMP also granted a positive opinion for the informed consent application Neparvis (sacubitril / valsartan) for the treatment of chronic heart failure with reduced ejection fraction. In an informed consent application, reference is made to an authorised medicine and the marketing authorisation holder of the reference medicine has given consent to the use of their dossier in the application procedure. The reference product for Neparvis is Entresto.

A generic medicine, Palonosetron Accord (palonosetron), received a positive opinion from the Committee for the prevention of nausea and vomiting associated with chemotherapy.

Three recommendations on extensions of therapeutic indications

The Committee recommended extensions of indications for Halaven, Humira and Opdivo.

Re-examination procedure concluded

The CHMP concluded a re-examination procedure, issuing a final negative opinion for Dropcys (mercaptamine). A questions-and-answers document on this opinion is available below.

Review on vancomycin

The CHMP started a review of medicines containing the antibiotic vancomycin as part of its efforts to update product information of older antibacterial agents in the context of the fight against antimicrobial resistance. The revision of product information for critically important antibiotics is considered an important way of promoting appropriate use to ensure they remain available to EU patients.

Review on Symbioflor 2

The Committee started a review of the medicine Symbioflor 2 (Escherichia coli bacteria), which is authorised in some Member States of the EU for treating diseases affecting the stomach and gut including irritable bowel syndrome.

Review of medicines for which studies have been conducted at Alkem BE, India

The CHMP also started a review of medicinesfor which studies have been conducted at the Alkem BE site in Mumbai, India. This follows a good clinical practice (GCP) inspection of this site which raised concerns regarding study data used to support the marketing authorisation applications of some medicines in the EU.

Posted on the EMA website on 1 April 2016