Regulation & Policy

It is not realistic to expect increased investment in medicines research in the EU or quicker access to new treatments for its citizens if member states insist on the lowest possible prices and highest government rebates, according to Nathalie Moll, Director General of Efpia, the European Federation of Pharmaceutical Industries and Associations. Ms Moll’s comments follow the publication of Efpia’s annual WAIT (Waiting to Access Innovative Therapies) report, which showed a picture of widening inequality among member states.

A provisional agreement by political authorities in the EU to support proposed legislation to improve the production and supply of critical medicines in the event of a disruption has been welcomed by the European Medicines Agency. Called the Critical Medicines Act, the legislation would spell out procedures for doing regular assessments of supply chains for the availability of essential medicines, identify vulnerabilities, and take steps to correct them. The EU has compiled a list of over 400 medicines which qualify as core products.

A gene therapy developed to treat hearing loss has been given an accelerated approved by the US Food and Drug Administration following the results of an ongoing single-arm trial. Developed by Regeneron Pharmaceuticals Inc, Otarmeni (lunsotogene parvec) was approved on 23 April and is the first gene therapy authorised for a genetic hearing loss. The disorder, OTOF-related hearing loss, is an ultra-rare condition affecting about 50 children born every year in the US. It is caused by variants in the OTOF gene and the lack of a functional otoferlin protein.

Baxfendy (baxdrostat), a small molecule drug in-licensed and developed by AstraZeneca Plc, has been approved by the US Food and Drug Administration to treat adults with hypertension – a disease estimated to affect 1.4 billion people worldwide and a leading risk factor for cardiovascular disease and premature death. Announced on 18 May, the approval is for the use of Baxfendy in combination with other antihypertensive medicines to lower blood pressure in adults who are not adequately controlled.

Marty Makary, the British-American surgeon and public health researcher, has resigned as commissioner of the US Food and Drug Administration after serving in the post for just over a year. He will be replaced, on an interim basis, by Kyle Diamantas, the agency’s deputy commissioner for food. The resignation was reported by Politico on 12 May and later confirmed by President Donald Trump to reporters the same day ahead of his trip to China. 

China’s regulatory authority, the National Medical Products Administration, has approved Blenrep (belantamab mafodotin), a treatment for multiple myeloma which is a relatively rare blood cancer but with a global prevalence. In China, it is now the only B-cell maturation antigen (BMCA) targeting second-line therapy for the disease. “Blenrep provides a new and needed mechanism of action in therapy,” the developer GSK Plc said in a statement on 20 April. Blenrep is an antibody-drug conjugate consisting of a humanised antibody conjugated to a cytotoxic agent via a non-cleavable linker.

Marty Makary, commissioner of the US Food and Drug Administration, has invited developers of testosterone replacement therapies for men to approach the agency to discuss a possible augmentation for their products. Specifically, this means pursuing a potential new indication for low libido in men with idiopathic hypogonadism, or sexual disfunction without a known cause. “New and emerging data suggest there may be an opportunity to help men suffering from symptoms that significantly affect quality of life,” he said in a statement issued on 16 April.

A veterinary vaccine using RNA technology and originally developed by Merck & Co has received a positive opinion from the European Medicines Agency on the basis of multiple, positive studies in cats. The vaccine, Nobivac NXT HCPChFeLV, contains live attenuated strains of five feline viruses, one of which is a strain of the feline leukaemia virus. This virus strain suppresses the immune system and is a common cause of cancer in cats. It is species specific and does not affect humans.

A small peptide drug in development at Oculis Holding AG of Switzerland has received regulatory designations from agencies in both Europe and the US for its promise as a treatment for optic neuritis, a disease that can seriously impair vision. Called privosegtor, the candidate drug activates pathways in the brain in order to promote the survival of neurons after an inflammatory attack. Optic neuritis can be caused by autoimmune diseases such as multiple sclerosis, or viral infections such as mumps, measles or the flu, according to the American Academy of Ophthalmology. 

A German company developing cell therapies to treat autoimmune diseases has been accepted into the European Medicines Agency’s support scheme PRIME, a regulatory pathway for small and medium-sized enterprises developing drugs for serious medical needs. The Heidelberg-based company, TolerogenixX GmbH, has received PRIME designation for its lead cell therapy candidate MIC-Lx for the treatment of immuno-suppression in live donor kidney transplantation.