Company Product Description Indication Status
Alexion Pharmaceuticals Inc., of Boston Ultomiris (ravulizumab) Monoclonal antibody targeting C5 Atypical hemolytic uremic syndrome EMA's Committee for Medicinal Products for Human Use adopted a positive opinion, recommending marketing authorization for patients with a body weight of 10 kg or above who are complement inhibitor treatment-naïve or have received Soliris (eculizumab) for at least 3 months and have evidence of response to eculizumab
Bristol Myers Squibb Co., of New York CC-486 Cytidine nucleoside analogue Maintenance treatment of adult patients in remission with acute myeloid leukemia FDA accepted the NDA and granted the application priority review; a PDUFA date is set for Sept. 3, 2020
Bristol Myers Squibb Co., of Princeton, N.J., and  Acceleron Pharma Inc., of Cambridge, Mass. Reblozyl Luspatercept Adults with anemia in beta-thalassemia and myelodysplastic syndromes EMA's Committee for Medicinal Products for Human Use adopted a positive opinion, which will now be reviewed by the EC
Essa Pharmaceuticals Inc., of Vancouver, British Columbia EPI-7386 Inhibits the N-terminal domain of the androgen receptor Prostate cancer FDA has notified the company that it may proceed with its proposed clinical investigation; Essa previously announced on March 30, 2020, that it had filed an IND application 
Galectin Therapeutics Inc., of Norcross, Ga. Belapectin (GR-MD-02) Galectin-3 inhibitor Prevention of esophageal varices in patients with nonalcoholic steatohepatitis cirrhosis Submitted to the FDA its protocol for a seamless adaptively designed phase IIb/III study, the NASH-RX trial, evaluating the safety and efficacy of belapectin
Genmab A/S, of Copenhagen, and Janssen Biotech Inc., a unit of New Brunswick, N.J.-based Johnson & Johnson Darzalex Faspro (daratumumab and hyaluronidase-fihi) Subcutaneous CD38-directed antibody Multiple myeloma FDA approved BLA for subcutaneous formulation for treatment of adults: in combination with bortezomib, melphalan and prednisone in newly diagnosed patients who are ineligible for autologous stem cell transplant (ASCT); in combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for ASCT and in patients with relapsed or refractory multiple myeloma who have received at least 1 prior therapy; in combination with bortezomib and dexamethasone in patients who have received at least 1 prior therapy; and as monotherapy, in patients who have received at least 3 prior lines of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent
Novan Inc., of Morrisville, N.C. SB-206 (berdazimer sodium) Nitric oxide-releasing macromolecule Molluscum contagiosum infection Received meeting minutes from the April 1, 2020, type C meeting with the FDA, and based on the guidance, the company is preparing to conduct 1 additional pivotal trial; the FDA provided guidance with regard to the study design and expectations for a future NDA submission
Pierre Fabre SA, of Castres, France Braftovi Encorafenib BRAFV600E-mutant metastatic colorectal cancer EMA's Committee for Medicinal Products for Human Use adopted a positive opinion for Braftovi in combination with cetuximab for the treatment of adult patients
PTC Therapeutics Inc., of South Plainfield, N.J. Gene therapy Gene therapy Aromatic L-amino acid decarboxylase deficiency Expects to file BLA with FDA in second half of 2020, a delay due to COVID-19
Sosei Heptares, part of Sosei Group Corp., of Tokyo, and Novartis AG, of Basel, Switzerland Enerzair Breezhaler (QVM-149) Indacaterol acetate, glycopyrronium bromide and mometasone furoate  Uncontrolled asthma EMA's Committee for Medicinal Products for Human Use recommended approval 
Telix Pharmaceuticals Ltd., of Melbourne, Australia TLX591-CDx 68 Ga-PSMA-11 injection Prostate cancer imaging Submitted an MAA in the EU
United Therapeutics Corp., of Silver Spring, Md. Trevyent (treprostinil) Preservative-free, parenteral formulation of prostacyclin analogue Pulmonary arterial hypertension Disclosed in its first-quarter earnings that FDA issued complete response letter (CRL) in April 2020; company has 1 year from date of CRL to resubmit the NDA
Vertex Pharmaceuticals Inc., of Boston Kalydeco (ivacaftor) Affects the gating of cystic fibrosis transmembrane conductance regulator Cystic fibrosis Received a positive opinion from EMA's Committee for Medicinal Products for Human Use for the label extension to include children and adolescents with CF between the ages 6 months and 18 years with the R117H mutation in the CFTR gene

Notes

For more information about individual companies and/or products, see Cortellis.

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