Regulation & Policy

The European Medicines Agency has given a conditional marketing authorisation to a gene therapy for haemophilia B which is a one-time treatment for the rare inherited blood disorder. Announced on 31 May, the decision is for fidanacogene elaparvovec, an adeno-associated virus based therapy that is designed to deliver a functional copy of the Factor 9 gene into the body enabling patients to produce Factor 9 protein themselves. The developer is Pfizer Inc which received US regulatory approval for the same therapy in late April.

The US Food and Drug Administration has expanded the permitted medical use of a gene therapy for Duchenne muscular dystrophy (DMD), making it available to patients who are both ambulatory and non-ambulatory. DMD is a genetic disorder characterised by a progressive deterioration of muscle due to abnormalities in, or the absence of, dystrophin protein which helps keep muscle cells intact.

Formycon AG of Germany has started clinical development of a candidate biosimilar of Keytruda (pembrolizumab), the checkpoint inhibitor for melanoma and other cancers that has revolutionised medical practice by blocking the interaction between two proteins and activating the immune system to kill cancer cells. To date, Keytruda has been approved for 48 medical indications.

Blincyto (blinatumomab), a bispecific antibody first approved for cancer in 2014, has received a new indication from the US Food and Drug Administration, extending its coverage for the treatment of B cell acute lymphoblastic leukaemia (B-ALL). B-ALL, an aggressive leukaemia, is the most common type of acute lymphoblastic leukaemia.
The new indication is for the treatment of adults and children from the age of one month for CD19 positive Philadelphia chromosome-negative B cell precursor acute lymphoblastic leukaemia. 

Donanemab, an Alzheimer’s disease drug developed by Eli Lilly and Co, is set for a review by the US Food and Drug Administration in the wake of a recommendation by the agency’s outside panel of advisers on 10 June that the drug is both safe and effective. Neither the FDA nor Lilly would comment to MedNous on a date for the final review. Nevertheless analysts who followed discussions by the panel – the Peripheral and Central Nervous System Drugs Advisory Committee – expect that whatever the timing, a formal approval is likely.

The Roche Group has received a ‘breakthrough therapy designation’ from the US Food and Drug Administration for a new breast cancer treatment intended for patients with a PIK3CA mutation. The PIK3CA gene makes an enzyme which is involved in many important functions in a cell. Mutations can cause the enzyme to become overactive and cancer cells to grow. 

The World Health Organization has designated the European Medicines Regulatory Network, a group of institutions that include the European Medicines Agency, as a WHO Listed Authority. The list, which was launched in March 2022, essentially gives the WHO access to the expertise of qualified regulators across the globe to help it improve the availability of safe and effective vaccines and medicines to people across geographies. This includes conducting evidence-based medicine assessments in situations where this expertise might not otherwise be available.

A new bispecific antibody drug has been approved for extensive-stage small cell lung cancer, marking an advance in the treatment of a disease with high relapse rates and very few therapy options. The drug, Imdelltra (tarlatamab), was given an accelerated approval by the US Food and Drug Administration on 16 May following a Phase 2 trial in patients with small cell lung cancer who had failed two or more prior lines of treatment. The developer is Amgen Inc.

Small and medium-sized enterprises (SMEs) saw a 33% increase in the success rate for their marketing authorisation applications in 2023, according to a new report by the European Medicines Agency. SMEs are broadly defined as companies with a staff headcount of between 10 and 250 people and a turnover ranging from less than €2 million to up to €50 million. Despite their small size, they represent the backbone of the European life science industry with activity in nine distinctive sectors. There were 1,925 registered SMEs on 31 December 2023. 

The US Food and Drug Administration has approved a new treatment for uncomplicated urinary tract (UTI) infections in women. Approximately one-half of all women experience at least one type of infection at least once in their lifetime. The drug, Pivya (pivmecillinam) is a narrow spectrum antibiotic which has been shown to be effective against infections caused by susceptible isolates of Escherichia coli, Proteus mirabilis and Staphylococcus saprophyticus.