Company Product Description Indication Status
Aadi Bioscience Inc., of Pacific Palisades, Calif. Nab-sirolimus (sirolimus albumin-bound nanoparticles for injectable suspension) mTOR inhibitor Advanced malignant PEComa (perivascular epithelioid-cell tumor) Initiated rolling NDA to the FDA seeking approval for the rare form of sarcoma driven by mTOR activation; submission to be completed in the fourth quarter of 2020
Allergan, a unit of North Chicago-based Abbvie, and Molecular Partners AG, of Zurich, Switzerland Abicipar pegol DARPin therapy Wet age-related macular degeneration FDA issued completed response letter to BLA, indicating the rate of intraocular inflammation observed following administration of 2 mg/0.05 mL results in unfavorable benefit-risk ratio
Ascendis Pharma A/S, of Copenhagen Transcon hGH (lonapegsomatropin) Long-acting, once-weekly prodrug of human growth hormone Pediatric growth hormone deficiency Submitted BLA to FDA
Axsome Therapeutics Inc., of New York AXS-05 Oral NMDA receptor antagonist Alzheimer’s disease agitation FDA granted breakthrough therapy designation
Bristol Myers Squibb Co., of New York, and Acceleron Pharma Inc., of Cambridge, Mass. Reblozyl (luspatercept) Erythroid maturation agent Anemia European Commission approved for use in adults with transfusion-dependent anemia due to very low-, low- and intermediate-risk myelodysplastic syndromes with ring sideroblasts, who had unsatisfactory response or are ineligible for erythropoietin-based therapy, and for adults with transfusion-dependent anemia associated with beta-thalassemia
Celltex Inc., of Houston AdMSCs Autologous adipose tissue-derived mesenchymal stem cells COVID-19 FDA cleared IND for phase II study testing safety and prophylactic efficacy; trial will enroll 200 patients who have never been infected with COVID-19
Chiasma Inc., of Needham, Mass. Mycapssa (octreotide) Oral somatostatin analogue Acromegaly Approved by FDA for use as long-term maintenance treatment in patients who have responded to and tolerated treatment with octreotide or lanreotide
Citius Pharmaceuticals Inc., of Cranford, N.J. Induced mesenchymal stem cells Induced mesenchymal stem cells COVID-19 Received written response from FDA regarding pre-IND to test iMSCs for treating and reducing severity of acute respiratory syndrome in COVID-19; agency said firm could apply for fast track designation and provided CMC requirements for proposed trials; company plans to initiate actions on FDA’s recommendations and follow up with IND application under the Coronavirus Treatment Acceleration Program
Daiichi Sankyo Co. Ltd., of Tokyo Pexidartinib Oral small molecule inhibiting CSF1R Severe tenosynovial giant cell tumor EMA’s Committee for Medicinal Products for Human Use adopted negative opinion on MAA for treating this subset of adults
Eisai Co. Ltd., of Tokyo Equfina (safinamide mesylate) Selective MAO-B inhibitor Parkinson’s disease Eisai Korea Inc. subsidiary received marketing approval in South Korea for treating idiopathic Parkinson's disease as adjunctive therapy with levodopa-containing products in patients with end of dose motor fluctuations
ERC Belgium SA, of Isnes, Belgium Sitoiganap (ERC-1671) Immunotherapy based on freshly extracted tumor cells and lysates Recurrent glioblastoma Submitted MAA to EMA seeking conditional approval
Gilead Sciences Inc., of Foster City, Calif. Remdesivir COVID19 non-structural protein 8 modulator/replicase polyprotein 1AB inhibitor; RNA polymerase COVID-19 EMA’s Committee for Medicinal Products for Human Use recommended conditionally approving the drug for use in patients, 12 and older, who require oxygen support
Hansa Biopharma AB, of Lund, Sweden Idefirix (imlifidase) Cleaves IgG-antibodies Kidney transplant EMA’s Committee for Medicinal Products for Human Use adopted positive opinion, recommending conditional approval for the desensitization treatment of highly sensitized adult kidney transplant patients with a positive crossmatch against an available deceased donor; endorsement by the European Commission expected in the third quarter of 2020
Novartis AG, of Basel, Switzerland Cosentyx (secukinumab) Inhibits interleukin-17A Moderate to severe plaque psoriasis EMA’s Committee for Medicinal Products for Human Use adopted positive opinion for approval in treating children and adolescents, ages 6 to under 18
Novartis AG, of Basel, Switzerland Xiidra (lifitegrast) Small-molecule integrin antagonist Moderate to severe dry eye disease Withdrew EMA MAA
Novartis AG, of Basel, Switzerland Xolair (omalizumab) Anti-IgE antibody Severe chronic rhinosinusitis with nasal polyps EMA’s Committee for Medicinal Products for Human Use recommended approval as add-on therapy with intranasal corticosteroids (INCs) to treat adults for whom therapy with INC does not provide adequate disease control
Pfenex Inc., of San Diego Livogiva (PF-708) Biosimilar to teriparatide injection Forsteo Osteoporosis EMA’s Committee for Medicinal Products for Human Use recommended approval, including for use in postmenopausal women with osteoporosis who are at high risk for having bone fractures
Philogen SpA, of Siena, Italy Nidlegy (bifikafusp alfa and onfekafusp alfa) Combination of immunocytokines Skin cancer Swissmedic cleared approval for phase II study in patients with locally advanced, not metastatic nonmelanoma skin cancer
Samsung Bioepis Co. Ltd., of Incheon, Korea Aybintio (bevacizumab biosimilar) Anti-VEGF antibody Multiple cancers EMA’s Committee for Medicinal Products for Human Use recommended approval for use in same types of cancer as reference bevacizumab in EU, including metastatic carcinoma of the colon or rectum, metastatic breast cancer, non-small-cell lung cancer, advanced and/or metastatic renal cell cancer, epithelial ovarian, fallopian tube and primary peritoneal cancer and cervical cancer
Sarepta Therapeutics Inc., of Cambridge, Mass. Casimersen (SRP-4045) Phosphorodiamidate morpholino oligomer Duchenne muscular dystrophy Completed submission of rolling NDA to FDA seeking accelerated approval for use in DMD patients who have genetic mutations amenable to skipping exon 45 of the Duchenne gene
Vertex Pharmaceuticals Inc., of Boston Kaftrio (ivacaftor/tezacaftor/elexacaftor) Combination of CFTR potentiator and 2 CFTR correctors Cystic fibrosis EMA’s Committee for Medicinal Products for Human Use recommended approval for use in a combination regimen with Kalydeco (ivacaftor) 150 mg to treat patients, 12 and older, with 1 F508del mutation and 1 minimal function mutation or 2 F508del mutations in the CFTR gene
Zogenix Inc., of Emeryville, Calif. Fintepla (fenfluramine) Antiseizure drug Dravet syndrome FDA approved for use in treating seizures associated with Dravet syndrome in patients, 2 and older

Notes

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

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