Regulation & Policy

Switzerland-based MetrioPharm AG has received a rare paediatric disease designation from the US Food and Drug Administration for its candidate therapy for Duchenne muscular dystrophy (DMD). The drug, MP1032, is a small molecule immune modulator that targets dysregulated mitochondrial metabolism in macrophages. In a preclinical study it was able to increase muscle strength comparable to the corticosteroid prednisolone, but without the corticosteroid’s serious side effects.

Sarepta Therapeutics Inc disclosed top-line results on 30 October from a Phase 3 confirmatory study of its gene therapy for Duchenne muscular dystrophy (DMD) which showed statistical significance for key secondary endpoints, but failed to meet the primary endpoint on a measure of motor function. The drug, Elevidys (delandistrogene moxeparvovec), was given an accelerated approval on 22 June 2023 based on a surrogate endpoint. The newest study was intended to confirm this clinical benefit.

Bimzelx (bimekizumab), a monoclonal antibody that inhibits two cytokines driving inflammation, has been approved in the US for the treatment of moderate to severe plaque psoriasis. The Food and Drug Administration cleared the drug for use on 18 October, more than a year after issuing a complete response letter to the developer, UCB SA of Belgium. The CRL was related to certain “pre-approval inspection observations,” according to the company.

The European Medicines Agency has given a positive opinion to a new drug for Duchenne muscular dystrophy (DMD), a genetic disorder primarily affecting males, which can lead to progressive muscle degeneration and weakness. The developer is Santhera Pharmaceuticals AG of Switzerland. The drug, Agamree (vamorolone), is a small molecule anti-inflammatory medicine that is being positioned as an alternative to corticosteroids, the current standard of care.

Pfizer Inc has received regulatory approval in the US for a personalised, or precision, medicine for lung cancer that demonstrated efficacy in a single-arm trial in both treatment naïve and previously treated patients with BRAF-V600E mutant metastatic non-small cell lung cancer. The medicine, Braftovi (encorafenib) and Mektovi (binimetinib), is a combination therapy that was first approved in 2018, but has since been updated. The newest approval includes two companion diagnostics that can identify BRAF V600E mutations in plasma or tumour tissue.

A diagnostic test for cancer was approved by the US Food and Drug Administration on 30 September which will help physicians detect hundreds of genetic variants associated with a person’s elevated risk of developing certain malignancies. Produced by Invitae Corp, the test also identifies potentially cancer-associated hereditary variants in individuals who have already been diagnosed with cancer.

GSK Plc has received US approval for Ojjaara (momelotinib), a Janus kinase inhibitor for the treatment of myelofibrosis patients with anaemia. The authorisation is based on positive results from two Phase 3 trials both of which were designed to show a reduction in manifestations of the disease. Myelofibrosis is a rare blood cancer that can lead to severely low blood counts, including anaemia and thrombocytopaenia.

In an article published on www.mednous.com on 21 August 2023 entitled SSI Strategy buys NDA Group, the leadership of the new group has been corrected to show that Douglas Locke, Chief Executive Officer of SSI Strategy, has been named CEO of the combined group. A corrected version follows.

A vaccine to prevent lower respiratory tract disease in infants which is caused by the respiratory syncytial virus (RSV) was approved by the Food and Drug Administration on 21 August – the second indication for this product in the US. Abrysvo has also been approved to prevent the same respiratory disease in people 60 years and older. Under the new indication, it will be administered as a single dose injection to pregnant women.

Ipsen SA has received Food and Drug Administration approval to market Sohonos (palovarotene) in the US for the treatment of fibrodysplasia ossificans progressiva (FOP), a rare genetic disease that causes connective tissue such as muscle and tendons to gradually turn into bone. This leads to a progressive loss of mobility and function for those affected. The approval was issued on 16 August and was based on the results of a Phase 3 trial of patients with the disease.