Regulation & Policy

From the personal computer to the iPhone, digital technologies have long been a feature of global communications. More recently, they have also begun to alter the landscape in healthcare. This was one of the themes addressed by Zaïde Frias of the European Medicines Agency to attendees of the Medtech & Pharma Platform virtual conference on 22 October. Ms Frias is head of the digital business transformation task force at the EMA, one of four groups set up by the agency in early March to prepare for new medical technologies.

The anti-viral treatment Veklury (remdesivir) has been authorised in the US for the treatment of patients with Covid-19 who require hospitalisation – the first coronavirus treatment to receive a full Food and Drug Administration approval. Announced on 22 October, the decision covers the drug’s use for patients 12 years and older. It follows the agency’s more restrictive emergency use authorisation (EUA) for Veklury, issued in May, which will now only cover the drug’s use in young children.

The European Medicines agency is recommending conditional approval of Tecartus (autologous anti-CD19-transduced CD3+ cells) for the treatment of relapsed or refractory mantle cell lymphoma. The chimeric antigen receptor (CAR) T cell therapy is the third medicine of its type to be reviewed by the agency. It follows the 2018 approvals of Kymriah for leukaemia and lymphoma and Yescarta for lymphoma.

The US Food and Drug Administration has given its first approval to a treatment for Ebola virus, a potentially fatal disease affecting people in the Democratic Republic of the Congo (DRC) and several other African countries. The treatment, Inmazeb, is a mixture of three monoclonal antibodies which bind to the glycoprotein on the surface of the virus, blocking its attachment to cell receptors and preventing its entry into cells.

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.