Regulation & Policy

A new triple combination therapy for cystic fibrosis, targeting the most common genetic mutation behind the disease, has been approved by the US Food and Drug Administration. Trikafta (elexacaftor/ivacaftor/tezacaftor) is now available to patients 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This is said to represent about 90% of all patients with the disease.

The first vaccine for the active immunisation of individuals at risk of infection from the Ebola virus is being recommended for use in countries affected by the disease including the Democratic Republic of Congo where a public health emergency has been declared by the World Health Organization.

The European Medicine Agency’s chief scientific committee has issued a positive opinion for Evenity (romosozumab) for the treatment of severe postmenopausal osteoporosis after initially rejecting the drug on the basis of cardiovascular safety concerns.

Denmark-based Bavarian Nordic A/S has won approval for Jynneos, a vaccine to prevent smallpox and monkeypox diseases for people at high risk of infection or in cases of a public health emergency. The development of Jynneos is the result of a 15-year partnership between Bavarian Nordic and the US government. The vaccine will become part of the US strategic national stockpile, which is the country’s largest supply of potentially life-saving medicines.

Regulatory agencies in the US, Canada and Australia have jointly approved a new treatment for endometrial cancer, the most common cancer of the female genital tract. The approval is the first of a new collaboration under which the Food and Drug Administration, Health Canada and the Australian Therapeutic Goods Administration review drug applications concurrently and issue a simultaneous decision afterwards.

The European Medicines Agency and the US Food Administration issued separate warnings on 13 September about ranitidine medicines, one of which is Zantac, which are widely used to treat heartburn and stomach ulcers. This follows tests that showed some products contained an impurity called N-nitrosodimethylamine (NDMA), classified as a probable carcinogen.

The European Medicines Agency has issued a positive opinion for the personalised therapy Xospata (gilteritinib) to treat acute myeloid leukaemia (AML) following approvals for the same drug in both the US and Japan. The developer is Tokyo-based Astellas Pharma Inc.

Xospata is being recommended as a monotherapy for the treatment of adult patients with relapsed or refractory AML with a FLT3 gene mutation. The positive opinion will need to be adopted by the European Commission before Xospata can be marketed.

Margrethe Vestager has been nominated for a second term as EU competition commissioner in a re-make of the union’s executive body that will see women take 13 out of the 27 top jobs and a 28-year old Lithuanian assume responsibility for the environment and oceans.

Over the past decade, the European Medicines Agency and the US Food and Drug Administration have stepped up their collaborations with the result that more than 90% of the marketing authorisation decisions made by the two organisations are aligned. This is the conclusion of an analysis conducted by the EMA and FDA, which compared outcomes on 107 new medicine applications between 2014 and 2016. While both agencies evaluate applications independently, they nevertheless are coming to many of the same conclusions.

A new antibiotic which has shown activity against pneumonia was cleared for marketing by the US Food and Drug Administration on 19 August. The safety and efficacy of Xenleta (lefamulin) were demonstrated in two clinical trials of 1,289 patients with community acquired bacterial pneumonia.