The EMA’s human medicines committee (CHMP) has recommended eight medicines for approval in its last meeting in London, two thereof orphan medicines. Three got conditional marketing authorisation.
The Committee recommended granting a conditional marketing authorisation for Ondexxya (andexanet alfa), Portola Pharmaceutical Inc's first-in-class antidote for patients taking the bloodthinners apixaban or rivaroxaban. Andexanet alfa reverses life-threatening uncontrolled bleeding.
The Committee adopted a positive opinion for Palynziq (pegvaliase), medicine for patients with phenylketonuria, a rare inherited metabolic disease. BioMarin International Ltd's orphan drug is a PEGylated recombinant phenylalanine ammonia lyase enzyme that converts phenylalanine to ammonia and trans-cinnamic acid, which are then eliminated from the body primarily by liver metabolism.
The CHMP recommended granting a conditional marketing authorisation for Waylivra (volanesorsen), the first medicine for the treatment of familial chylomicronaemia syndrome, another orphan disease that prevents the body from breaking down fats. In August 2018, market approval of Akcea Therapeutics Inc‘s antisense inhibitor of APOCIII designed to lower high blood triglyceride levels was delayed. It received a complete response letter from the FDA.
Pfizer Europe was granted a recommendation for conditional marketing authorisation by the CHMP for Lorviqua (lorlatinib)for the treatment of patients with anaplastic lymphoma kinase-positive advanced non-small cell lung cancer. The protein kinase inhibitor (L01XE36) inhibits autophosphorylation of ALK, ALK-mediated phosphorylation of downstream signalling proteins and proliferation of ALK-dependent cancer cells.
The Committee recommended granting GlaxoSmithKline a marketing authorisation under exceptional circumstances for FDA-approved neuraminidase inhibitor Dectova (zanamivir) for the treatment of complicated and potentially life-threatening influenza.
Abbvie Deutschland’s Skyrizi (risankizumab), a mAb targeting the p19 subunit of the human pro-inflammatory cytokine interleukin 23 (IL-23), received a positive opinion for the treatment of moderate to severe psoriasis.
The Committee also recommended to approve Teva BV‘s generic medicine Pazenir (paclitaxel), for the treatment of metastatic breast cancer and non-small cell lung cancer.
Sanofi‘s Zynquista (sotagliflozin), intended as an add-on therapy to insulin for certain patients with type 1 diabetes, received a positive opinion from the CHMP though ongoing debates on the side effect profile of the SGLT1 /2 inhibitor at the FDA.
