Swissmedic Authorizes Three Orphan Drugs for Rare Diseases: Breyanzi, Alhemo, and Ekterly

Swissmedic has recently authorized three distinct orphan drugs for the treatment of rare, life-threatening diseases. The regulatory decisions encompass an indication extension for the CAR-T cell therapy Breyanzi, an expanded indication for the haemophilia treatment Alhemo, and the initial authorization of the hereditary angioedema therapy Ekterly.

The Swiss agency authorized an indication extension for Breyanzi (active substance: lisocabtagene maraleucel) on 19 February 2026. The product is now approved for the treatment of adults with relapsed or refractory mantle cell lymphoma (MCL) who have received at least two prior lines of systemic therapy, including a BTK inhibitor. MCL is a rare and aggressive lymph node cancer. Breyanzi is a CD19-directed CAR-T cell therapy using genetically modified T cells from the patient. The efficacy was demonstrated in the TRANSCEND-MCL study, which included 88 adults. The study found that the majority of patients responded to treatment, with a large proportion achieving complete remission. The most common side effects include cytokine release syndrome, reduced blood cell counts, fatigue, and headache. Breyanzi was first authorized in Switzerland in February 2022 and had a previous extension for follicular lymphoma in June 2025.

An indication extension for Alhemo (active substance: concizumab) was authorized on 15 December 2025. Alhemo is a subcutaneous injection for preventing or reducing bleeding in adults and adolescents aged 12 and over with severe haemophilia A without inhibitors or moderate-to-severe haemophilia B without inhibitors. The drug works by blocking a natural factor to activate blood clotting despite factor deficiencies. A clinical study showed that prophylactic use of Alhemo significantly reduced both spontaneous and accident-related bleeding compared to needs-oriented treatment without prophylaxis. The most common adverse effect is injection site reactions, with severe side effects including allergic reactions and blood clots. Alhemo was initially authorized in August 2023 for haemophilia patients with inhibitors.

Ekterly (active substance: sebetralstat) was authorized on 17 September 2025 for treating acute attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 and older. HAE is a rare inherited disease causing painful, potentially life-threatening swellings. The oral tablets contain sebetralstat, which blocks plasma kallikrein activity to reduce bradykinin formation and halt attack progression. The authorization was made as part of the Access Consortium joint initiative, involving collaborative assessment with regulatory authorities in Australia, Singapore, and the United Kingdom.

All three medicinal products have been designated as orphan drugs due to their use in treating rare, life-threatening conditions. Swissmedic first authorized Breyanzi in 2022, while Alhemo received its initial approval in 2023. Ekterly represents a new therapeutic option for HAE patients in Switzerland.