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CHMP Positive Opinions | EMA Drug Approvals | European Commission | 2023 | IPHARMACENTER

Updated: May 8

APRIL 2023

Amicus Therapeutics' Opfolda (miglustat) received a positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) to treat Late-Onset Pompe Disease.

The European Commission approval is anticipated in the third quarter of 2023. Opfolda is given in combination with Pombiliti (cipaglucosidase alfa). The company stated that once the approval of Opfolda is received, the drug will be available as a combination with Pombiliti.

Opfolda is an enzyme stabilizer given in combination with long-term enzyme replacement therapy, Pombiliti (cipaglucosidase alfa).

The approval was based on the Phase 3 PROPEL pivotal study, a randomized trial in LOPD patients who are ERT-naive and ERT-experienced.

Roche announced that the CHMP recommended approving Columvi (glofitamab) to treat adults with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) post two or more lines of therapy.

The approval was based on the Phase I/II NP30179 study, demonstrating the efficacy in patients with R/R DLBCL. Results showed that Columvi given as a fixed course had induced a complete response (CR) in 35.2% of people and an overall response (OR) in 50% of people. Within patients who achieved CR, 74.6% of patients had experienced a response at 12 months. The duration of the response still needs to be reached.

The common adverse events are cytokine release syndrome, neutropenia, anemia, and thrombocytopenia.

Bristol Myers Squibb (BMS) received CHMP positive opinion for CAMZYOS (mavacamten) to treat symptomatic obstructive hypertrophic cardiomyopathy (HCM).

The positive recommendation was based on two Phase 3 trials, EXPLORER-HCM and VALOR-HCM.

The Phase 3 EXPLORER-HCM trial evaluated efficacy in patients with symptomatic obstructive HCM. The Phase 3 VALOR-HCM study was conducted in patients with symptomatic obstructive HCM who met the 2011 ACC/AHA or 2014 ESC guideline criteria. The drug met all the primary and secondary endpoints in both studies.

GlaxoSmithKline's (GSK) vaccine candidate for the respiratory syncytial virus (RSV) in older adults has received a positive opinion from the CHMP.

The vaccine candidate, known as RSVPreF3, is a protein-based vaccine that targets RSV