The key facts

  • Iqirvo (elafibranor) and Hympavzi (marstacimab) have been registered — both are classified as original drugs
  • Hympavzi is indicated for patients with severe hemophilia A or B without inhibitors, aged 12 and older with a body weight of 35 kg or more
  • In October, primary biliary cholangitis was added to the list of rare diseases — the list contains 306 entries

Two registrations for orphan disease patients

Russia's Ministry of Health has registered two new drugs for the treatment of rare (orphan) diseases, the ministry's press service reported. These are Iqirvo with the active ingredient elafibranor, intended for adults with primary biliary cholangitis, and Hympavzi with the active ingredient marstacimab. The ministry emphasized that both products are original (innovative) medicines and contain active ingredients synthesized and registered for the first time.

Who the drugs are intended for

Primary biliary cholangitis is a rare chronic disease in which the immune system damages the small bile ducts of the liver, impairing bile flow; Iqirvo is intended specifically for such adult patients. Hympavzi is used to prevent and reduce the frequency of bleeding episodes in severe hemophilia A or B without inhibitors of clotting factors — in patients aged 12 and older weighing at least 35 kg. Hemophilia is a hereditary blood clotting disorder in which even minor injuries can cause prolonged bleeding, and prophylactic therapy is aimed at reducing the number of such episodes.

Context: the list of rare diseases and the share of domestic drugs

The registration took place against the backdrop of recent changes in the regulation of orphan diseases: in October, the Ministry of Health added primary biliary cholangitis to the Russian list of rare diseases, which now contains 306 entries after the update. Later, Deputy Health Minister Sergey Glagolev said that about 40% of the drugs registered in the country for orphan diseases are domestic, and Russian pharmaceutical companies are engaged not only in analogues but also in their own developments. At the same time, the ministry separately noted that the registration of a drug does not in itself mean it will immediately appear in all medical facilities and pharmacies.