CHMP meeting highlights: February 2024

At its February 2024 meeting, the EMA’s human medicines committee (CHMP) recommended the extension of marketing authorisations for six treatments, and positive opinions for the approval of ten medicines.

The committee adopted positive opinions for two vaccines intended for active immunisation against the H5N1 subtype of influenza A virus:

• Celldemic (zoonotic influenza vaccine (H5N1)(surface antigen, inactivated, adjuvanted, prepared in cell cultures)), is intended for immunisation during outbreaks of influenza coming from animals, including when public health authorities anticipate a possible pandemic
• Incellipan (pandemic influenza vaccine (H5N1) (surface antigen, inactivated, adjuvanted, prepared in cell cultures).

The CHMP recommended granting a conditional marketing authorisation for Filspari* (sparsentan) for the treatment of primary immunoglobulin A (IgA) nephropathy.

A new treatment for ALS

A marketing authorisation for the new treatment Qalsody* (tofersen), which Biogen licensed from Ionis Pharmaceuticals, was recommended by the CHMP for exceptional circumstances in adults with the rare disease amyotrophic lateral sclerosis (ALS). It is indicated in those with a mutation in the superoxide dismutase 1 (SOD1) gene.

EMA highlighted that the antisense oligonucleotide therapy works by binding to the mRNA of the SOD1 gene. This decreases the production of SOD1 protein and is expected to improve the symptoms of ALS.

Clinical data showed approximately 60 percent reductions in plasma NfL concentrations in patients given Qalsody compared to placebo. This indicated reduced neuronal injury, EMA noted. Patients also experienced improvement in their physical abilities, according to the data.

Treating NSCLC

Tizveni (tislelizumab) received a positive opinion from the CHMP for the treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) in adults. According to BeiGene, the Marketing Authorisation Application (MAA) for NSCLC is based on results from three Phase III studies. For example, first-line combination therapy evaluated tislelizumab in advanced squamous NSCLC in the Phase III RATIONALE 307 study.

A novel oral treatment

Moreover, the CHMP gave a positive opinion for Voydeya* (danicopan), the first oral treatment against residual haemolytic anaemia in patients with paroxysmal nocturnal haemoglobinuria, a rare genetic blood disorder.

According to the EMA, the active substance of Voydeya is danicopan. This is a complement inhibitor immunotherapy that prevents red blood cells from being destroyed, and thus helps to relieve the symptoms of PNH.

This recommendation is mainly based on Phase III study results in patients given C5 complement inhibitors ravulizumab or eculizumab. After 12 weeks, patients treated with danicopan had a clinically-relevant increase in haemoglobin (an average of 2.35 g/dl) compared to those given placebo, EMA confirmed.

Other positive opinions by the CHMP

• Zynyz* (retifanlimab) as a treatment for Merkel cell carcinoma, a type of skin cancer
• Pyzchiva (ustekinumab), a biosimilar used to treat plaque psoriasis
• The generic medicine Apremilast Accord (apremilast), for psoriatic arthritis, psoriasis and Behçet’s disease, a rare type of inflammatory disease
• Generic medicine Nintedanib Accord (nintedanib) in adults with idiopathic pulmonary fibrosis, other chronic fibrosing interstitial lung diseases with a progressive phenotype, and systemic sclerosis associated interstitial lung disease.

Medicine indication extensions recommended by the CHMP 

The committee recommended extensions of indications for six EU-approved medicines, including: CARVYKTI^®▼ (ciltacabtagene autoleucel; cilta-cel)*, Keytruda and Reblozyl*.

Carvykti

According to the Janssen Pharmaceutical Companies of Johnson & Johnson, the CHMP recommended cilta-cel as a treatment for adults with relapsed and refractory multiple myeloma (RRMM).

EMA explained that the CAR-T therapy is indicated in individuals who have had least one prior therapy, including an immunomodulatory agent (IMiD) and a proteasome inhibitor (PI), have demonstrated disease progression on the last therapy, and are refractory to lenalidomide.

The innovative treatment is the first CAR-T therapy to receive a positive CHMP opinion for adults with relapsed and refractory multiple myeloma (RRMM).

“Early resistance to standard treatments is becoming more common in patients with lenalidomide-refractory multiple myeloma, highlighting a need for new options earlier in the course of treatment,” stated Dr Edmond Chan, MBChB, Senior Director, EMEA Therapeutic Area Lead Haematology, Janssen-Cilag Limited, a Johnson & Johnson Company. The CHMP’s recommendation “recognises the potential of cilta-cel to significantly improve outcomes for eligible patients with relapsed and refractory multiple myeloma, as early as after first relapse.”

The CHMP recommendation is supported by data from the CARTITUDE-4 study. This is the first randomised Phase III study evaluating the efficacy and safety profile of cilta-cel versus pomalidomide, bortezomib and dexamethasone (PVd) or daratumumab, pomalidomide and dexamethasone (DPd) in relapsed and lenalidomide-refractory multiple myeloma who received one to three prior lines of therapy, according to the Janssen Pharmaceutical Companies of Johnson & Johnson.

*These products were designated as an orphan medicine during their development.

CHMP meeting highlights: January 2024