European Regulators Endorse Multiple New Drugs, Including Breakthrough Treatments for Rare Diseases

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of ten new pharmaceutical products, marking significant advancements in the treatment of various conditions, including rare diseases and cancer.

Ionis and Otsuka's Dawnzera Breaks New Ground in HAE Treatment

Ionis Pharmaceuticals' RNA-targeted therapy for hereditary angioedema (HAE), Dawnzera, has received a positive opinion from the CHMP. Developed in partnership with Otsuka Pharmaceuticals for the European and Asia Pacific markets, Dawnzera demonstrated an impressive 81% reduction in HAE attacks compared to placebo in phase 3 trials. This novel treatment, which recently gained FDA approval, is poised to become the first RNA-targeted therapy for HAE in Europe, offering a unique dosing schedule and the convenience of pre-filled pens for at-home injections.

Otsuka's chief executive in Europe hailed the CHMP opinion as a "key milestone in advancing access to potentially life-changing treatments for the HAE community." The genetic condition, affecting approximately 1 in 50,000 people worldwide, causes recurrent attacks of severe swelling in various parts of the body.

Eli Lilly's Inluriyo and Other Oncology Advancements

In a significant development for breast cancer treatment, Eli Lilly's oral selective estrogen receptor degrader (SERD) Inluriyo received CHMP endorsement. The drug is intended for patients with locally advanced or metastatic breast cancer harboring an activating ESR1 mutation. If approved by the European Commission, Inluriyo will be indicated for use in patients who have experienced disease progression following endocrine-based therapy.

This recommendation places Inluriyo as only the second oral SERD to gain regulatory approval in Europe or the United States, following Italy's Menarini's breakthrough in 2023.

Diverse Range of Therapies Endorsed by CHMP

The CHMP's latest meeting yielded positive opinions for a variety of innovative treatments:

• Sanofi's Teizeild, a disease-modifying therapy for Type 1 diabetes, received endorsement under CHMP's priority medicine (PRIME) pathway.
• Fondazione Telethon's Waskyra, the first gene therapy for Wiskott-Aldrich syndrome, marking a milestone as the first non-profit organization to shepherd a drug from research to regulatory approval.
• BioNet Europe's new whooping cough (pertussis) vaccine.
• Curium Romania SRL's radionuclide generator.

Additionally, the committee recommended two biosimilars and two generics, including versions of Sanofi's long-acting insulin Lantus, Amgen's bone medications Prolia and Xgeva, and Astellas and Pfizer's prostate cancer drug Xtandi.

The CHMP's endorsements now move to the European Commission for final approval decisions, potentially expanding treatment options for patients across Europe in the near future.