Regulation & Policy

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.

A new chimeric antigen receptor (CAR) T cell therapy has been approved for cancer – only the third such therapy to be authorised by the US Food and Drug Administration. The drug is Tecartus (brexucabtagene autoleucel), a cell-based gene therapy for the treatment of mantle cell lymphoma, a rare form of B cell non-Hodgkin’s lymphoma which usually occurs in middle-aged or older adults.

AstraZeneca Plc has announced the US approval of its triple-combination therapy, Breztri Aerosphere, for the maintenance treatment of patients with chronic obstructive pulmonary disease (COPD). The drug combines budesonide, an inhaled corticosteroid, with glycopyrrolate, a long-acting muscarinic antagonist, and formoterol fumarate, a long-acting beta2 agonist.

The checkpoint antibody Keytruda has been approved as a first-line treatment for a certain type of metastatic colorectal cancer, bringing the number of US cancer indications for the drug up to 28. The approval was based on the results of a Phase 3 trial in which Keytruda was shown to reduce the risk of disease progression or death by 40% compared with chemotherapy, the current standard of care. Patients in the trial had metastatic microsatellite instability-high or mismatch repair deficient colorectal cancer.

The US Food and Drug Administration has approved a new treatment for seizures associated with Dravet syndrome, but with a warning that the drug is associated with valvular heart disease and pulmonary arterial hypertension. The drug, Fintepla (fenfluramine), is an amphetamine derivative.

After reviewing data from a US trial of remdesivir, the European Medicines Agency is recommending that the anti-viral treatment be given a conditional marketing authorisation to treat patients in the EU with COVID-19-associated pneumonia who require supplemental oxygen. The recommendation now goes to the European Commission for approval.