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CHMP Opinions | EMA drug approvals

Updated: Jun 27, 2023

March 2021 CHMP Positive Opinion


Verastem’s Copiktra (duvelisib) received CHMP positive opinion for treating CLL and FL

CHMP gave a positive opinion for Verastem’s Copiktra (duvelisib) for treating relapsed or refractory chronic lymphocytic leukemia (CLL) and refractory follicular lymphoma (FL).

In the clinical trials, Copiktra had shown improvement in progression-free survival versus ofatumumab in patients who received two prior treatments. Respiratory tract infections, neutropenia, anemia, thrombocytopenia were the common adverse reported with the drug.

Copiktra is available in 15 mg and 25 mg capsules. The drug acts by inhibiting phosphatidylinositol 3-kinase p110δ (PI3K-δ) and PI3K-γ.

The drug has been previously approved by the US FDA and is marketed by Secura Bio.






CHMP gave a positive opinion for Janssen’s Ponvory to treat multiple sclerosis


CHMP gave a positive opinion for Janssen’s Ponvory (ponesimod) to treat adult patients with relapsed multiple sclerosis.

The positive opinion was based on the Phase 3 OPTIMUM study, a multicentre, randomized, double-blind, parallel-group study, which is intended to demonstrate the superiority of Ponvory over Aubagio (teriflunomide).

Ponvory demonstrated superiority versus Aubagio in reducing the annual relapsed rate (ARR) by 30.5%, the primary endpoint. It showed superiority in the secondary endpoint (unique active lesions).





Efmody (hydrocortisone) received a positive opinion to treat congenital adrenal hyperplasia (CAH)


Diurnal Europe BV received a positive opinion for Efmody (hydrocortisone) for treating congenital adrenal hyperplasia.



GSK’s Benlysta received a positive opinion for active lupus nephritis


GlaxoSmithKline announced that CHMP gave a position opinion for Benlysta (belimumab) to treat adult patients with active lupus nephritis.

The approval is based on Phase 3 BLISS-LN study, with Primary Efficacy Renal Response at two years (or 104 weeks) as the primary endpoint. Benlysta has demonstrated significant improvement compared to the standard of care plus placebo combination.

In December 2020, GSK received the FDA approval for Benlysta to treat patients with lupus nephritis.



Roche’s Tecentriq received a positive opinion for NSCLC label extension

CHMP gave a positive opinion for Tecentriq (atezolizumab) to treat NSCLC patients. The drug got a positive opinion as a monotherapy to treat adult patients with metastatic NSCLC, PD-L1 expression ≥ 50% tumor cells (TC) or ≥ 10% tumor-infiltrating immune cells (IC) not having either EGFR or ALK mutant.




FEBRUARY 2021



Evrysdi received a positive opinion for spinal muscular atrophy

Evrysdi received positive opinion for treating patients with 5q SMA in patients two months of age and older.

The positive opinion is based on two clinical studies: FIREFISH in symptomatic Type 1 infants aged 2 to 7 months and SUNFISH in symptomatic Type 2 and 3. In SUNFISH, adult patients with SMA Types 2 and 3 SMA were included.


In the FIREFISH study, efficacy was evaluated based on the ability to sit without support for at least five seconds using the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition. 93% were alive, and 85% were event-free after 12 months of treatment with Evrysdi.

In the SUNFISH study, a double-blind, placebo-controlled trial included patients with Types 2 or 3 SMA. Motor Function Measure 32 (MFM-32) at 12 months was used to evaluate the motor function. There was a clinically meaningful and significant improvement in motor function compared to placebo.




BioCryst’s Orladeyo received a positive opinion for the prevention of recurrent attacks of hereditary angioedema (HAE)

Orladeyo (berotralstat) received the CHMP positive opinion for preventing the recurrent attacks of hereditary angioedema (HAE).

The positive opinion is based on the APeX-S trial. In APeX-2, met primary endpoint compared to placebo.

The product has received the orphan designation in Europe.

The drug has been previously approved in the US. BioCryst Pharmaceuticals also applied a marketing authorization application (MAA) for the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA).



GSK’s dostarlimab got positive opinion for recurrent or advanced endometrial cancer

Dostarlimab, an anti-programmed death-1 (PD-1) monoclonal antibody, received positive opinion for treating women with mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) recurrent or advanced endometrial cancer.

The positive opinion is based on GARNET study, ORR was 42% and a disease control rate was 58%.



SEPTEMBER 2020


MenQuadfi and Supemtek

In Europe, CHMP gave a positive recommendation for Sanofi’s vaccine candidates, MenQuadfi and Supemtek.

MenQuadfi is indicated for the active immunization of individuals from the age of 12 months and older against meningococcal ACWY disease. The approval is based on the clinical trials, which demonstrate an immune response against serotype A, C, W, and Y.

Sanofi submitted seven double-blinded, randomized, multicenter, Phase 2, and Phase 3 clinical trials, demonstrating the immune response of the vaccine in toddlers of 12 months and older.

The vaccine is expected to available in the European market from the beginning of next year.



Sanofi also received a positive recommendation for another vaccine, Supemtek (quadrivalent recombinant influenza vaccine), development for the prophylaxis of influenza in adults 18 years and older.

The approval is based on Phase 3 randomized trials involving more than 10,000 patients. The risk of influenza was reduced by more than 30% in adults age 50 years and older.

Supemtek relies on recombinant manufacturing technology. The drug is already approved in the U.S., and Sanofi has supplied more than 10 million doses of vaccine.


OLUMIANT FOR ATOPIC DERMATITIS

Eli Lilly received the CHMP positive opinion for baricitinib for treating adults with moderate to severe atopic dermatitis.

Olumiant (baricitinib) is the first JAK inhibitor to receive the CHMP positive opinion for atopic dermatitis.

The CHMP positive opinion was received based on Phase 3 BREEZE-AD clinical trials. BREEZE-AD1 and BREEZE-AD2 were designed to investigate the efficacy and safety of baricitinib in patients with moderate to severe atopic dermatitis as monotherapy, BREEZE-AD4 was intended to demonstrate the efficacy of baricitinib in combination with corticosteroids in patients who have failed or intolerant or contraindicated to corticosteroids.

The product was prior approved in more than 70 countries for rheumatoid arthritis.



LYNPARZA

AstraZeneca and MSD’s Lynparza have received the CHMP positive opinion for treating patients with metastatic castration-resistant prostate cancer (mCRPC).

AstraZeneca and MSD have presented the PROfound Phase III trial to support the CHMP positive opinion. The progression-free survival was considered as the primary endpoint; the progression-free survival was 7.4 months in the Olaparib group compared to 3.6 months in the control group. The median overall survival was 18.5 months in the Olaparib group versus 15.1 months in the placebo group.

Lynparza has received the approval for metastatic prostate cancer in May 2020.

 

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