The European pharmaceutical landscape is set to expand with innovative orphan drug treatments following positive recommendations from the Committee for Medicinal Products for Human Use (CHMP). Two groundbreaking therapies for rare diseases have cleared a crucial regulatory hurdle, paving the way for their anticipated European approval within the next three months.
Revolutionary RNA Treatment for hATTR Amyloidosis
Alnylam Pharmaceuticals' Onpattro (patisiran) represents a significant advancement in treating hereditary transthyretin-mediated amyloidosis (hATTR) in adults with stage 1 or stage 2 polyneuropathy. This rare and life-threatening condition stems from mutations in the transthyretin (TTR) gene. Onpattro employs innovative small interfering RNA (siRNA) technology, designed to interrupt TTR protein production in the liver by targeting specific genetic material.
"We are ready to launch patisiran following the EC decision, and hope that it will help to meet the pressing need for new treatment options for patients living with hATTR amyloidosis in Europe," stated Theresa Heggie, head of Europe at Alnylam Pharmaceuticals. Notably, Onpattro will become the second approved treatment for hATTR amyloidosis, following Akcea and Ionis' recently authorized Tegsedi (inotersen).
Expanding Treatment Options for Cystic Fibrosis
The CHMP has also endorsed Vertex's Symkevi (tezacaftor/ivacaftor), expanding the company's portfolio of cystic fibrosis treatments. The therapy, designed to be used in combination with Kalydeco, targets patients carrying one copy of the F508del mutation along with one of 14 mutations resulting in residual CFTR activity. This approval builds upon Vertex's established treatments, including Kalydeco (ivacaftor) and Orkambi (ivacaftor+lumacaftor).
Broader Impact on Rare Disease Treatment Landscape
The committee's recommendations extended beyond these orphan drugs. Additional positive opinions were issued for:
- Pierre Fabre's melanoma treatments Braftovi (encorafenib) and Mektovi (binimetinib)
- AstraZeneca's Imfinzi (durvalumab) for lung cancer
- Lilly's Verzenios (abemaciclib) for breast cancer
- Two biosimilar versions of Amgen's Neulasta (pegfilgrastim)
However, the committee delivered negative opinions for Bristol-Myers Squibb's Opdivo (nivolumab) and Yervoy (ipilimumab) combination as a first-line kidney cancer treatment, and Amgen's Blincyto for minimal residual disease in B-precursor acute lymphoblastic leukemia patients.
These recommendations reflect the ongoing evolution of targeted therapies for rare diseases and demonstrate the rigorous evaluation process for new treatments in the European market. The anticipated approvals will provide healthcare providers with expanded options for treating patients with these challenging conditions.
