The agency that recommends what drugs should be approved in Europe has thrown its support behind five new ones, including a gene therapy for hemophilia, a combination treatment for liver cancer and a medicine for a rare disorder known as Pompe disease.
While the European Commission still needs to authorize these treatments, the nods from the European Medicines Agency and its drug review committee put them a step closer to market.
If approved, CSL Behring’s Hemgenix would become the first hemophilia gene therapy commercially available in Europe. Hemgenix is specifically designed to treat the less common “B” form of the rare bleeding disorder. It’s also one of the world’s most expensive therapies, carrying a list price of $3.5 million in the U.S., where it received marketing clearance last month.
For years, hemophilia patients have kept their disease in check by receiving infusions of the blood clotting proteins they’re missing. Though effective, these infusions can still be burdensome and are often expensive.
Hemophilia has therefore become a prime target for genetic medicine developers, which seek to treat the disease with therapies that offer a one-time “fix.” Hemgenix, for example, uses an engineered virus to deliver functional copies of the gene responsible for making a certain clotting protein.
The EMA based its approval recommendation off of results from two clinical trials that enrolled 57 hemophilia B patients and found that the vast majority of them sustained positive effects from Hemgenix over multiple years. Patients treated with the therapy experienced reductions in the number of bleeding episodes each year, from over 4 down to about 1.5, and nearly all of them were able to discontinue the routine, preventive infusions they needed before.
Like Hemgenix, AstraZeneca’s immunotherapy Imjudo recently secured approval in the U.S.
Imjudo blocks the effects of a protein called CTLA-4, which is well-known among cancer researchers because it helps regulate the immune system.
In October, the Food and Drug Administration OK’d a combination of Imjudo and another AstraZeneca immunotherapy, Imfinzi, for adults with a certain, common type of liver tumor that can’t be removed through surgery. Then, in November, it was approved again, in combination with Imfinzi and platinum-based chemotherapy for adults with advanced non-small cell lung cancer.
The EMA supports Imjudo’s clearance for these indications in Europe as well — though, in non-small cell lung cancer, the recommendation is for “Tremelimumab AstraZeneca,” which has the same active ingredient.
Meanwhile, Amicus Therapeutics has run into repeated delays getting the FDA to approve its medicine for Pompe disease, Pombiliti. Pompe is an inherited, often fatal illness characterized by weakened muscles and impaired heart and lung function.
Authorization in Europe may provide a lift to Amicus and its rare disease business, which is currently anchored by a single product, Galafold, that brought in around $240 million in net sales between January and September of this year.
The EMA said it adopted a positive opinion on Pombiliti because of the drug’s ability improve the motor function in patients with late-onset Pompe disease when used in combination with another medication known as miglustat.
Along with Hemgenix, Imjudo and Pombiliti, the EMA also recommended approval for a generic version of Biogen’s blockbuster drug for multiple sclerosis, Tecfidera.