EMA has recommended granting a marketing authorisation in the European Union (EU) for Kimmtrak (tebentafusp), a monotherapy for the treatment of adult patients with uveal melanoma, a rare type of eye cancer.
EMA’s human medicines committee (CHMP) reviewed the application for marketing authorisation under an accelerated timetable to enable faster patient access to this medicine in view of the high unmet medical need.
The CHMP based its recommendation on data from a randomised Phase 3 pivotal study and a supportive study. The pivotal study included 378 previously untreated patients with advanced uveal melanoma, of whom 252 were randomly selected to receive tebentafusp and 126 were in the control group and received one of three already established therapies for the condition (dacarbazine, ipilimumab or pembrolizumab). Tebentafusp was administered to patients via intravenous infusion. The main measure of effectiveness was overall survival (how long the patients lived). The study showed that Kimmtrak prolonged patients’ lives: the median overall survival was 21.7 months for patients receiving tebentafusp and 16 months for patients in the control group.
The most common side effects observed in clinical trials were skin rashes, fever and itching.
What is uveal melanoma
Uveal melanoma is a rare and aggressive disease in which cancer cells form in the tissues of the eye. Signs of uveal melanoma include blurred vision or a dark spot on the iris. Patients with uveal, or ocular, melanoma often have a poor prognosis as the disease can resist treatments and spreads quickly through the body with the liver being the most frequent site of metastasis (disease spreading to other parts of the body). Once the disease has spread, many patients survive less than a year.
Currently, the most widely used first-line treatment options for non-metastatic disease for this cancer are surgery, radiation therapy, and enucleation (procedure by which the entire eye is removed). The condition is found primarily in the population with light skin pigmentation and light-coloured eyes. It is estimated that uveal melanoma affects between five and eleven patients per million.
Tebentafusp, the active substance of Kimmtrak, is a type of treatment called a bispecific fusion protein. It works by helping immune cells to get close enough to the cancer cells to attack them. The treatment can be used in adult patients who are human leukocyte antigen (HLA)-A*02:01-positive and have unresectable (cannot be removed surgically) or metastatic uveal melanoma.
The opinion adopted by the CHMP is an intermediary step on Kimmtrak’s path to patient access. The opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, decisions about price and reimbursement will take place at the level of each Member State, taking into account the potential role/use of this medicine in the context of the national health system of that country.