Patient voice overcomes EU ‘no’ for UCB/Amgen’s osteoporosis drug

UCB/Amgen’s osteoporosis drug Evenity (romosozumab) looks set for approval in Europe after regulators finally recommended it, following a successful appeal against a rejection with help from doctors and patients.

The positive opinion by the CHMP follows the FDA’s approval of the drug in April, after a rejection in 2017.

It’s notoriously difficult to overturn a negative opinion from Europe’s CHMP scientific committee, which makes regulatory recommendations that the European Commission nearly always rubber stamps after a few months.

UCB reckons that only 20-25% of appeals against CHMP rejections are successful, and last year only one out of five appeals against negative opinions for new drug applications succeeded.

Dr Pascale Richetta, executive vice president and head of the Bone Patient Value Unit at UCB, said the CHMP’s initial concern was about the cardiovascular safety signal that emerged in the ARCH trial.

In the US, Amgen and UCB got around the initial rejection with a request for a narrower indication in women at high risk of fracture, instead of in the population of all postmenopausal women.

In Europe, the companies have also gone for the more focused indication, but needed testimony from two patients and doctors who highlighted the pressing need for a new therapy to win over the initially sceptical CHMP.

Under the European Medicines Agency’s rules, appeals must not include any new data, and as such companies can only present new analyses and new arguments in favour of approval.

Richetta told pharmaphorum in a telephone interview that the safety issue from ARCH was “based on small numbers”, and that the company may “not have articulated the unmet medical need and how much the benefit was unprecedented."

“They (the CHMP) looked at further analysis of the data, and also took additional advice from patient representatives and experts on osteoporosis and cardiovascular disease.”

“The patients articulated the unmet medical need, and that one fracture brings risk of another fracture.”

“We are enjoying the remarkable achievement of the regulatory team. We are really proud to have done that for Europe.”

UCB and Amgen will get an equal share of profits from the drug worldwide, and the focus now shifts to launches in Europe following a likely approval later this year.

Aside from the US, the drug is also approved in markets including Japan, Australia, Canada, and South Korea, with other countries likely to follow.

Evenity is important to Amgen as a successor to its blockbuster osteoporosis drug Prolia/Xgeva (denosumab).

Prolia, a RANK inhibitor, is one of Amgen’s top-selling drugs with sales of $2.3 billion last year, but will start losing patent protection in Europe in 2022 and in the US in 2025.