CHMP backs Sanofi’s all-oral sleeping sickness drug

A new drug that could revolutionise the treatment of millions of people with trypanosomiasis – commonly known as sleeping sickness – has been backed for approval in the EU.

Sanofi’s Fexinidazole Winthrop (fexinidazole) now has a positive opinion from the EMA’s Committee for Medicinal Products for Human Use (CHMP) and – assuming full approval in the coming weeks – could be the first all-oral drug available for the disease.

Trypanosomiasis is usually fatal if left untreated, but current therapies are either a painful series of intramuscular injections with pentamidine of patients are in the early stages of the disease, or oral nifurtimox and intravenous infusion of eflornithine in cases that have advanced to the stage that it involves the central nervous system.

“Fexinidazole Winthrop, as exclusively oral treatment regimen for the disease, could potentially allow quicker and wider access to treatment because distribution and administration of tablets is easier,” according to the EMA. It’s taken as a 10-day, once-daily treatment course.

Currently the bulk of the burden of trypanosomiasis -which is spread by tsetse fly bites – is felt in the Democratic Republic of the Congo, with the remainder located in bordering central African countries, where the delivery of healthcare can be problematic. Current therapies require patients to be hospitalised.

The drug was developed in collaboration with the Drugs for Neglected Diseases initiative (DNDi) and has been reviewed by the EMA under the Article 58 mechanism, which gives the CHMP licence to assess and give opinions on medicines that are intended for use in countries outside the EU. Sanofi says it will start distributing fexinidazole in endemic countries next year.

CHMP round-up

Fexinidazole was one of four new medicines recommended for approval by the CHMP at its meeting last week.

Johnson & Johnson got a positive opinion on its non-metastatic castration-resistant prostate cancer (CRPC) drug Erleada (apalutamide), setting up EU approval to follow its FDA green light earlier in the year for adult patients with treatment-resistant tumours at high risk of spreading. Erleada is one of ten new drug candidates that J&J thinks could eventually make sales of more than $1bn a year and was the first drug to be registered on the strength of metastasis-free survival (MFS).

Macimorelin Aeterna Zentaris (macimorelin) was backed for the diagnosis of growth hormone deficiency in adults. German drugmaker Aeterna Zentaris' oral ghrelin agonist was approved by the FDA for the same indication in 2017 under the tradename Macrilen. According to the EMA it can diagnose GH deficiency “with high sensitivity and specificity in adult patients.”

Italy’s Recordati also got a positive opinion for Silodosin Recordati (siloosin) a generic of its own Urorec drug used to treat benign prostatic hyperplasia (BPH).

The CHMP also gave its blessing to various line extensions for already-market drugs. Among these, Novartis’ CDK4/6 inhibitor Kisqali (ribociclib) was recommended for the treatment of women with hormone receptor-positive, HER2-negative locally advanced or metastatic breast cancer in combination with fulvestrant as initial endocrine-based therapy as well as in women who have received prior endocrine therapy.

The panel reversed an earlier negative verdict on Amgen’s Blincyto (blinatumomab) for adults with Philadelphia-negative, CD19 positive B-cell precursor acute lymphoblastic leukaemia (ALL) in first or second complete remission with minimal residual disease greater than or equal to 0.1%, and also backed Roche’s MabThera (rituximab) for adults with severe, active granulomatosis with polyangiitis and microscopic polyangiitis in combination with corticosteroids.

Vertex Pharma got the okay to extend the use of its Orkambi (lumacaftor/ivacaftor) cystic fibrosis drug to include patients aged 2 to 5 years old who have two copies of the F508del mutation, while Horizon Pharma’s Ravicti (glycerol phenylbutyrate) got the go-ahead for se as an adjunctive therapy for patients with urea cycle disorders who can’t manage their condition with dietary interventions alone.