After FDA rejection, Gilead nears full EU nod for hep D drug

Gilead Sciences is on the brink of getting full approval for the first drug for hepatitis D virus (HDV) in the EU, after getting a positive opinion from the EMA’s human medicines committee.

The CHMP has recommended approval of Hepcludex (bulevirtide) for the treatment of adults with chronic HDV and compensated liver disease, which would upgrade an earlier conditional approval for the drug in 2020.

At the time, the EMA said it was concerned about limitations in the clinical data submitted in support of the drug – including a small number of patients in its supporting trials – but opted to give it the green light as there are limited treatment options for HDV patients. Current treatment with pegylated interferon alpha products – which takes 48 weeks to complete – isn’t very effective.

Gilead has since submitted 48-week results from its phase 3 MYR301 data, which showed that the antiviral drug achieved a significantly greater combined virological and biochemical response when compared to untreated patients.

HDV is unusual in that it exists only as a co-infection with hepatitis B virus (HBV) and occurs in around 5% of the estimated 257 million people around the world who are chronically infected with HBV – around 12 million people.

Numbers have declined in recent years because of HBV vaccination programmes, but there are still hotspots around the world. Infection is also serious, as having both HBV and HDV increases the risk of liver disease-related death and liver cancer, and mortality rates can be as high as 50% within five years, according to Gilead.

The recommendation is a boost for Gilead, which was knocked back last year when the FDA issued a complete response letter (CRL) for bulevirtide, apparently on concerns about the manufacture and delivery of the drug.

Gilead said at the time that the US regulator had not asked for any additional safety or efficacy trials, suggesting the delay could be short-lived. In its latest update, the company noted that it “remains in active discussions with the FDA, with the aim of bringing bulevirtide to people living with HDV in the US as soon as possible. “

Gilead reported $13 million of Hepcludex sales in the fourth quarter of 2022, but did not break out sales of the drug in its first-quarter update. Analysts have suggested that with US approval, sales of the drug could approach $250 million a year.

The company acquired bulevirtide as part of its €1.15 billion ($1.4 billion) takeover of Germany’s MYR in 2021.