CHMP Backs 13 New Medicines, Maintains Negative Opinion of Sarepta’s Exondys

Regulatory NewsRegulatory News | 21 September 2018 |  By 

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) on Friday recommended thirteen new medicines for EU approval and reaffirmed its negative opinion of Sarepta Therapeutics’ Duchenne muscular dystrophy drug Exondys (eteplirsen).
Among the thirteen drugs recommended for approval are three orphan drugs, three Neulasta (pegfilgrastim) biosimilars, two anti-cancer drugs and a new antibiotic.
The three orphan medicines include Spark Therapeutics’ gene therapy Luxturna (voretigene neparvovec), Bayer’s hemophilia A drug Jivi (damoctocog alfa pegol) and Kyowa Hakko’s Sézary syndrome treatment Poteligeo (mogamulizumab).
The two cancer treatments include Takeda’s second-line non-small cell lung cancer treatment Alunbrig (brigatinib), which was granted accelerated approval in the US last year, and Oasmia Pharmaceutical’s ovarian cancer drug Apealea (paclitaxel), which has not yet been submitted to the US Food and Drug Administration (FDA) for review.
According to CHMP, the newly recommended antibiotic, Rempex Pharmaceuticals’ Vabomere (meropenem trihydrate/vaborbactam), combines a beta-lactamase inhibitor and an already-approved carbapenem antibiotic to overcome certain carbapenem-resistant infections.
CHMP also recommended extending the indications for seven other drugs, including Ipsen’s Cabometyx (cabozantinib), Novartis’ Gilenya (fingolimod), Roche’s Actemra (tocilizumab) and Astellas’ Xtandi (enzalutamide).
Additionally, the committee says it will re-examine its recent decisions against extending the indications of Amgen’s Blincyto (blinatumomab) and Bristol-Myers Squibb’s Opdivo (nivolumab) and Yervoy (ipilimumab).
The decision to reject the Exondys, known as Exondys 51 in the US, comes two years after FDA’s controversial decision to grant the drug accelerated approval despite a negative advisory committee vote and objections from members of the review team.
CHMP initially rejected Sarepta’s marketing authorization application for Exondys in May, finding that the clinical studies submitted as evidence for the drug showed “no meaningful difference between Exondys and placebo in the 6-minute walking distance.”
After re-examining its decision at Sarepta’s request, the committee says it will uphold its initial decision to reject the drug.
“While largely anticipated, we are disappointed with the outcome of the CHMP re-examination and firmly believe that eteplirsen should be made available to patients in Europe, as it is in the United States,” said Sarepta President and CEO Doug Ingram.


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