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.
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.
It has been approved for patients who have relapsed, after other kinds of treatment, and targets CD19, an antigen expressed in all B cell lineage malignancies.
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.