Seven new medicines - including the first gene therapy for patients with an ultra-rare immune disorder - have been endorsed by regulatory advisors for approval in Europe. 

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended GlaxoSmithKline’s orphan-designated Strimvelis for the treatment of patients with adenosine-deaminase-deficient severe combined immunodeficiency (ADA-SCID), who have no matching donor for a stem cell transplant. 

Children born with this disorder - caused by a faulty gene inherited from both parents that stops the production of adenosine deaminase, an enzyme that a toxic substance called deoxyadenosine - have virtually no immunity to fight off everyday bacterial, fungal or viral infections.

Strimvelis was studied in a pivotal clinical trial involving 12 patients, all of which are still alive, with an average follow-up period of seven years. There is currently no authorised medicine to treat ADA-SCID in the EU.

Elsewhere, the CHMP issued a conditional marketing authorisation for Janssen-Cilag’s Darzalex (daratumumab) for the treatment of relapsed and refractory multiple myeloma. The monoclonal antibody has an orphan designation and was reviewed under EMA’s accelerated assessment scheme. 

Conditional approval allows the CHMP to recommend a medicine for marketing authorisation in the interest of public health where the benefit of its immediate availability to patients outweighs the risk inherent in the fact that additional data are still required.

In this case, the decision was based on two studies; in one, tumours shrank or could no longer be seen in 29 percent of patients taking the drug over an average of 7.4 months; and in the second, tumours shrank or could no longer be seen in 36 percent.

Janssen must provide results from two Phase III studies of Darzalex used in combination with standard treatments for the disease (lenalidomide/dexamethasone and bortezomib/dexamethasone) for regulators to consider a full approval.

Positive recommendations were also handed down for: Amicus Therapeutics’ Galafold (migalastat) for the treatment of Fabry diseaser; AstraZeneca's Pandemic influenza vaccine H5N1 MedImmune, indicated for the prevention of flu and intended for pandemic preparedness; Samsung Bioepis’ biosimilar monoclonal antibody Flixabi (infliximab), for rheumatoid arthritis, adult and paediatric Crohn's disease, ulcerative colitis, paediatric ulcerative colitis, ankylosing spondylitis, psoriatic arthritis and psoriasis; Novartis’ Neparvis (sacubitril/valsartan), for heart failure with reduced ejection fraction; and Accord Healthcare’s generic Palonosetron Accord (palonosetron), for the prevention of nausea and vomiting associated with chemotherapy.