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EMA Drug Approvals | CHMP Positive Opinions in October 2023 | Europe drug approvals | iPharmaCenter


Santhera Pharmaceuticals' Agamree received CHMP's positive opinion for DMD.

Santhera Pharmaceuticals has received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) recommending the approval of Agamree (vamorolone) to treat Duchenne muscular dystrophy (DMD) in children and adults aged four years and older.

Agamree could become the first drug fully endorsed by the EMA to treat DMD if approved. There is also anticipation of vamorolone's approval in the U.S., with the Prescription Drug User Fee Act (PDUFA) date set for October 26, 2023.

The positive recommendation from CHMP is based on clinical evidence from the pivotal VISION-DMD study, several open-label studies, an external comparator study, and various clinical pharmacology investigations.

The study successfully achieved its primary endpoint, which involved assessing the change in TTSTAND velocity from baseline to week 24 for the group receiving Agamree at a dose of 6 mg/kg per day compared to the placebo group. While the placebo group exhibited a relatively stable trajectory with a slight decline from their baseline measurements, the Agamree group receiving 6 mg/kg daily showed significant improvement as early as six weeks into the treatment.


CHMP gave a positive opinion to Pfizer's Elrexfio for multiple myeloma

The Committee for Medicinal Products for Human Use (CHMP) issued a positive recommendation for the conditional marketing authorization of Elrexfio, a medication designed to treat multiple myeloma. It is indicated in patients who were previously treated with three other therapies.

Its active ingredient, elranatamab, is a bispecific monoclonal antibody that targets the CD3 receptor found on the surface of T cells and BCMA expressed on plasma cells, including malignant multiple myeloma cells.

The key benefit of Elrexfio lies in its capacity to induce a partial or complete response in relapsed or refractory multiple myeloma patients, as demonstrated in a Phase 2 open-label study. Common side effects encompass cytokine release syndrome, anaemia, neutropenia, fatigue, upper respiratory tract infections, and reactions at the injection site.

This product received orphan designation during its development, and the EMA will review available information to determine if the orphan designation can be retained.


CHMP positive recommendation brings new treatment options for hyperargininemia patients.

The CHMP recommended granting marketing authorization under special circumstances for the medicinal product Loargys.

This product is designed to treat hyperargininemia, and the market authorization holder is Immedica Pharma.

Loargys will be accessible as a 5 mg/ml solution for injection or infusion. The active component in Loargys is pegzilarginase, classified under ATC code A16AB24, an enzyme that effectively reduces arginine levels in the bloodstream.

The primary advantage of Loargys is its capacity to alleviate the manifestations and clinical symptoms associated with arginase 1 deficiency (ARG1-D) compared to a placebo, as evidenced in a well-conducted multicenter, double-blind, placebo-controlled trial. The most commonly reported side effect observed during the trial was hypersensitivity.


CHMP gave a positive opinion for Veoza to treat hot flashes associated with menopause.

The Committee for Medicinal Products for Human Use (CHMP) issued a favourable opinion, recommending the authorization of Veoza, a medicinal product designed to treat hot flushes (vasomotor symptoms) associated with menopause.

Veoza falls under gynaecological products and will be provided as a 45 mg film-coated tablet. The active ingredient in Veoza is fezolinetant, a non-hormonal substance that can penetrate the blood-brain barrier and act upon the thermoregulatory centre of the hypothalamus.

The key benefit of Veoza is its capacity to decrease the occurrence and intensity of moderate to severe vasomotor symptoms, often known as hot flashes. This efficacy has been established through two 12-week, randomized, placebo-controlled, double-blind Phase 3 studies conducted with postmenopausal women.



BeiGene's Brukinsa received a CHMP position recommendation for relapsed or refractory follicular lymphoma.

BeiGene has received a positive opinion from the CHMP for approving Brukinsa (zanubrutinib), a Bruton's tyrosine kinase inhibitor (BTKi). The recommendation is for its use in combination with obinutuzumab as a treatment for follicular lymphoma who have undergone at least two prior lines of systemic therapy. It is recommended for relapsed or refractory forms of cancer in adult patients.

Compelling clinical trial data from the ROSEWOOD study and the BGB-3111-GA101-001 study support the CHMP's decision. The ROSEWOOD study is a Phase 2 study of Brukinsa combined with obinutuzumab, compared to obinutuzumab alone, involving 217 patients with R/R FL who had received a minimum of two prior lines of systemic therapy. In this randomized, open-label study, the Brukinsa plus obinutuzumab arm demonstrated an overall response rate of 69.0%, compared to 45.8% in the obinutuzumab arm, with approximately 20 months of median follow-up. The combination therapy was well-tolerated, with safety results consistent with previous studies of both medicines.

Brukinsa is already approved in the E.U. for specific indications, including chronic lymphocytic leukaemia and marginal zone lymphoma. Regulatory submissions for Brukinsa in R/R FL are also being reviewed by the U.S., China, Canada, Switzerland, and the United Kingdom authorities as part of the Access Consortium New Active Substance Work-sharing Initiative.


AstraZeneca's Imfinzi is approved as a standalone treatment for the initial therapy of advanced or inoperable hepatocellular carcinoma (HCC) in adults. Previously, it was approved for HCC in combination with tremelimumab.


CHMP recommended selecting Jemperli as a first-line treatment option for dMMR/MSI-H endometrial cancer.

GSK announced positive CHMP opinion recommending the approval of Jemperli (dostarlimab) in combination with carboplatin-paclitaxel (chemotherapy) as a first-line option for treating mismatch repair deficient (dMMR)/microsatellite instability-high (MSI-H) primary advanced or recurrent endometrial cancer. The drug, if approved, can be used in adults who are eligible for systemic therapy.

GSK's application for dostarlimab's authorization is based on RUBY/ENGOT-EN6/GOG3031/NSGO phase III trial (part 1 of the trial is basis for positive recommendation).

The study findings are derived from a substantial median follow-up duration of at least 25 months. Part 1 of the RUBY trial successfully met its primary endpoint by demonstrating investigator-assessed progression-free survival (PFS) in patients who received dostarlimab alongside carboplatin and paclitaxel within the dMMR/MSI-H population. This group reduced the risk of disease progression or death by 72%.

Additionally, in a pre-defined exploratory analysis of overall survival among the dMMR/MSI-H population, the combination of dostarlimab and chemotherapy led to reduction in the risk of death by 70% compared to chemotherapy alone.


Keytruda received CHMP positive opinion as a first-line therapy option for adult patients with GEJ adenocarcinoma.

Merck received a CHMP positive opinionfor Keytruda (pembrolizumab) to treat adult patients with locally advanced unresectable or metastatic HER2-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma. It is recommended as a first-line therapy for adults. This positive opinion recommends the approval of Keytruda, Merck's anti-PD-1 therapy, in combination with chemotherapy containing fluoropyrimidine and platinum.

The basis for this recommendation is the results from the Phase 3 KEYNOTE-859 trial. In this trial, combining Keytruda with chemotherapy showed a benefit in overall survival versus chemotherapy. Notably, this benefit was observed in patients whose tumours expressed PD-L1, with approximately 80% of the study's participants falling into this category.


Sanofi's Praluent received a positive CHMP opinion to treat pediatric patients. Praluent is approved for adults with primary hypercholesterolaemia, familial or non-familial, and those with mixed dyslipidaemia. It can be administered to pediatric patients aged eight years and older with heterozygous familial hypercholesterolaemia (HeFH) as a dietary adjunct if approved by EMA.



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