The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended 13 new medicines for approval, including three orphan drug candidates.
The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of 13 new medicines, including three orphan drug candidates, the agency announced on Sept. 21, 2018.
Among the recommendations for approval are Spark Therapeutics’ gene therapy, Luxturna (voretigene neparvovec), an orphan drug for treating inherited retinal dystrophy caused by RPE65 gene mutations, a rare genetic disorder which causes vision loss and usually leads to blindness, and Eli Lilly and Company’ s Emgality (galcanezumab), a monoclonal antibody for preventing migraine that belongs to a new class of medicines that block calcitonin gene-related peptide (CGRP) activity.
The committee also recommend granting marketing authorization for The Medicines Company’s new antibiotic, Vabomere (meropenem trihydrate / vaborbactam), for treating various severe infections in adults.
Two new cancer medicines, Takeda Pharmaceutical’s Alunbrig (brigatinib), for treating anaplastic lymphoma kinase-positive advanced non-small cell lung cancer, and Oasmia Pharmaceutical’s Apealea (paclitaxel), for treating ovarian cancer, were also recommended for approval.
The two other orphan medicines to receive a positive opinion are Bayer’s Jivi (damoctocog alfa pegol), for treating hemophilia A, and Kyowa Hakko Kirin's Poteligeo (mogamulizumab), for treating mycosis fungoides or Sézary syndrome. The committee also gave a positive opinion to Merck & Co.’s Delstrigo (doravirine / lamivudine / tenofovir disoproxil) and Pifeltro (doravirine), both for treating HIV-1 infection.
In addition, the committee also recommend approval of three biosimilars to Amgen’s Neulasta (pegfilgrastim) intended to reduce the duration of neutropenia and the incidence of febrile neutropenia due to chemotherapy: Mylan’s Fulphila (pegfilgrastim), Cinfa Biotech’s Pelmeg (pegfilgrastim), and Sandoz’s Ziextenzo (pegfilgrastim).
Finally, the committee granted a positive opinion for Camurus’ Buvidal (buprenorphine), a hybrid medicine for treating opioid dependence. Hybrid applications rely in part on the results of pre-clinical tests and clinical trials of a reference product and in part on new data.
Upon follow-up re-examination, CHMP also had the following negative recommendations on new medicines:
• Sarepta Therapeutics’ Exondys (eteplirsen), for which Sarepta requested a re-examination following the committee’s negative opinion adopted in May 2018. Upon consideration of this request, the committee confirmed its previous recommendation to refuse the granting of a marketing authorization. The drug was approved in the United States by FDA in September 2016.
• Amgen’s Blincyto (blinatumomab) and Bristol Myers Squibb’s Opdivo (nivolumab) and Yervoy (ipilimumab), for which the companies requested re-examination of the committee’s negative opinions adopted in July 2018. The CHMP will now re-examine the opinions and issue final recommendations.
The committee is also expanding its review of impurities in valsartan following the detection of very low levels of N-nitrosodiethylamine (NDEA) in another active substance, losartan, made by Hetero Labs in India. As a result of the detection of this impurity by German authorities, the review will now include medicines containing four other ‘sartans’, namely, candesartan, irbesartan, losartan, and olmesartan.
Applications for initial marketing authorizations for TMC Pharma Services’ Entolimod TMC (entolimod) and SciPharm Sàrl’s Treprostinil SciPharm Sàrl (treprostinil) have been withdrawn. Treprostinil SciPharm Sàrl was intended for the treatment of chronic thromboembolic pulmonary hypertension.
Source: EMA
Trump Directs FDA, EPA to Reduce Regulatory Barriers to Domestic Pharma Manufacturing
May 7th 2025The White House is instructing FDA to increase fees for and inspections of foreign drug manufacturing plants and reduce the time required to approve such sites that will be newly constructed in the US.
New Cancer Research and Treatment Center in Development in London
May 1st 2025Aviva Capital Partners and developer Socius are investing £1 billion in a 12-acre site to be located at the London Cancer Hub next to The Institute of Cancer Research, London and The Royal Marsden NHS Foundation Trust’s Sutton site.
Regeneron Treatment for Multiple Myeloma Gets Conditional Marketing Approval from EC
April 29th 2025The indication is specific to patients who have received at least three prior therapies, including a proteasome inhibitor, immunomodulatory agent, and anti-CD38 monoclonal antibody, and have demonstrated disease progression on the last therapy.