Dive Brief:
- Roche on Tuesday said its experimental medicine crovalimab showed promise in late-stage testing for a rare blood disorder, keeping the company on track to join a crowded market led by AstraZeneca’s top-selling drug Soliris.
- The drugmaker's Genentech unit tested the drug in people with paroxysmal nocturnal hemoglobinuria, or PNH, a condition in which the body’s “complement” system destroys defective red blood cells. In one study, crovalimab was assessed against Soliris on how well it prevented the loss of red blood cells and the resulting need for blood transfusions in patients with PNH.
- The company did not disclose detailed data, but noted China has already granted the drug priority review and added that submissions to other regulators around the world will follow. It plans to present detailed data from the studies at an upcoming medical meeting.
Dive Insight:
PNH has long been the domain of Alexion Pharmaceuticals, which developed Soliris and its successor drug Ultomiris for the disease and other complement-mediated conditions. They are now owned by AstraZeneca following its purchase of Alexion.
The disorder, which can leave patients anemic and at risk of blood clots, is considered a rare disease, and its treatments sell at very high prices. For AstraZeneca, Soliris and Ultomiris together generated revenue of about $4.3 billion in the first nine months of last year.
The large market has attracted competition. Apellis won approval of a rival drug in 2021. And last year, Novartis reported two trial wins for a potential competitor, leading its executives to predict peak sales above $3 billion.
AstraZeneca is also testing an experimental “add-on” drug for PNH that it said in November would soon be submitted for regulatory review.
Like Soliris and Ultomiris, Roche’s drug crovalimab blocks a protein known as C5 to calm the body’s complement system. Roche tested its drug, given by injection, against Soliris in a randomized study of around 200 participants with PNH who had not been previously treated with a complement inhibitor. It also ran a study testing the safety of switching PNH patients to crovalimab from other approved C5 inhibitors.
On an earnings call last week, Roche CEO Thomas Schinecker said with crovalimab “we believe, in terms of efficacy, we have differentiation” because the antibody uses a “recycling technology” from Japanese drugmaker Chugai, which is partially owned by Roche. “With that, we have a much higher level of activity because the antibodies can be in the body [and] reused multiple times.”