Introduction:

The Committee for Medicinal Products for Human Use (CHMP) held its February 2024 meeting with numerous noteworthy outcomes. CHMP assessments are based on extensive scientific data analysis. They assess innovative drugs quality, safety, efficacy, and risk-benefit ratio. Internal peer-review ensures committee opinions are accurate and valid. In this article we highlight the key takeaways and their outcomes.

Ten New Medicines for Approval

The CHMP meeting saw the recommendation of ten new innovative medicines for approval. Two of these were vaccines aimed at active immunisation against the H5N1 subtype of influenza A virus, more commonly known as avian influenza or bird flu. The vaccines, Celldemic and Incellipan, are designed for immunisation during outbreaks of influenza from animals, including potential pandemics.

Innovations in Disease Treatment

Other significant recommendations included Filspari for the treatment of primary immunoglobulin A nephropathy, a disease where the kidneys gradually cease functioning, and Qalsody, a new therapy for treating adult patients with amyotrophic lateral sclerosis (ALS), a rare and often fatal disease, causing muscle weakness and leading to paralysis.

Advancements in Cancer and Blood Treatment

The meeting also saw positive opinions in the oncology and haematology landscape, including a recommendation for Tizveni, a treatment for locally advanced or metastatic non-small cell lung cancer, and Zynyz, a treatment for Merkel cell carcinoma, a life-threatening skin cancer with poor outcomes with advanced disease. Furthermore, the CHMP endorsed Voydeya, the first oral treatment for residual haemolytic anaemia in patients with paroxysmal nocturnal haemoglobinuria, a rare genetic disorder that causes the immune system to prematurely break down red blood cells. Voydeya was classified as an orphan drug during its development phase.

Biosimilar and Generic Medicines Approved

The CHMP meeting also saw the approval of Pyzchiva, an ustekinumab biosimilar intended for the treatment of plaque psoriasis, psoriatic arthritis, ulcerative colitis, and Crohn’s disease. In addition, two generic medicines received a positive opinion from the committee: Apremilast Accord for the use in the treatment of psoriatic arthritis, psoriasis and Behçet’s disease and also Nintedanib Accord for adults with idiopathic pulmonary fibrosis, other chronic fibrosing interstitial lung diseases with a progressive phenotype, and systemic sclerosis associated interstitial lung disease.

Extensions of Therapeutic Indication

The CHMP meeting recommended extensions of indication for six medicines already authorised in the European Union, including Carvykti, Cibinqo, Kalydeco, Keytruda, Reblozyl, and Xromi. The applicants for Nezglyal (also initially an orphan drug) and Syfovre requested the January 2024 committee’s opinions reexamined, and final recommendations will be provided.

Conclusion:

The outcomes of the CHMP February 2024 meeting highlight the importance of a well-defined peer-review committee in the healthcare sector in order to evaluate and recommend new and innovative medicines. By ensuring an evidence-based process the CMHP strives to provide high level recommendations and allows for a thriving healthcare system which provides ample opportunity for innovation.