Regulation & Policy

Switzerland-based Santhera Pharmaceuticals AG has discontinued development of Puldysa (idebenone), its candidate therapy for Duchenne muscular dystrophy after an independent review of an ongoing Phase 3 trial concluded that the trial was unlikely to meet its primary endpoint. The Data and Safety Monitoring Board based its conclusion on an interim analysis of the study, SIDEROS, which was being conducted in Duchenne muscular dystrophy patients using concomitant glucocorticoids.

The European Medicines Agency has started a rolling review of a prospective vaccine for Covid-19 being developed by Oxford University and AstraZeneca Plc on the basis of preliminary data showing that it triggered the production of antibodies and T cells that target the virus. The agency announced the review on 1 October.

The US Food and Drug Administration has approved a new indication for Nucala (mepolizumab) for hypereosinophilic syndrome (HES), a group of rare blood disorders characterised by the overproduction of eosinophils, a type of white blood cell. It is the first approval for this patient group in nearly 14 years.

Prescription drugs sold in Canada at cheaper prices than in the US will be eligible for importation under a new rule adopted by the US Food and Drug Administration on 25 September. The rule implements an executive order issued by the White House in July.

The gene and cell therapy industry has significant potential. But this will only be realised if manufacturers share information about their technologies, enabling the advanced therapies to be produced at scale and marketed at affordable prices.

BioMarin Pharmaceutical Plc has received a complete response letter (CRL) from the US Food and Drug Administration saying it can’t approve the company’s gene therapy for haemophilia A without further data on its durable effect. The therapy, valoctocogene roxaparvovec, is intended as a one-time treatment for the blood clotting disorder.

An application from Gilead Sciences Inc to market filgotinib, a candidate treatment for rheumatoid arthritis, has been turned back by US Food and Drug Administration with a request for further safety data, the company announced on 19 August. Filgotinib, a JAK1 inhibitor, was discovered by Galapagos NV and is being jointly developed with Gilead.

A new therapy has been approved by the US Food and Drug Administration to treat a rare central nervous system (CNS) disorder known as neuromyelitis optica spectrum disorder – the third drug to be authorised for this indication in a little over a year. The drug, Enspryng (satralizumab), is a monoclonal antibody developed by Chugai Pharmaceutical Co Ltd, a member of the Roche group.

The European Commission has concluded an advance purchase agreement with AstraZeneca Plc for the supply of up to 400 million doses of a Covid-19 vaccine which it is developing with the University of Oxford. Announced on 14 August, the agreement covers the initial purchase of 300 million doses, with an option for an additional 100 million. The price was not disclosed. However in a separate announcement, AstraZeneca said it would supply the vaccine “in an eqitable manner at no profit during the pandemic.”

MorphoSys AG has secured regulatory approval from the US Food and Drug Administration for its first proprietary antibody therapeutic, Monjuvi (tafasitamab), to treat adult patients with relapsed or refractory diffuse large B cell lymphoma (DLBCL). The approval was based on data from the Phase 2 L-MIND study, a multicentre, single arm trial of Monjuvi in combination with lenalidomide, a drug for multiple myeloma. DLBCL is the most common type of non-Hodgkin lymphoma that can develop in the lymph nodes or any organ of the body.