Company Product Description Indication Status
Algernon Pharmaceuticals Inc., of Vancouver, British Columbia NP-120 (ifenprodil) NMDA receptor antagonist COVID-19 Requested end-of-phase II meeting with FDA following completion of phase IIb part of phase IIb/III study
Amydis Inc., of San Diego Retinal tracer Small-molecule diagnostic Parkinson's disease Completed pre-IND interaction with FDA, which concurred with specifications for drug substance and planned toxicology studies to support phase I/IIa development
Astrazeneca plc, of Cambridge, U.K., and Merck & Co. Inc., of Kenilworth, N.J. Selumetinib Dual MEK-1/MEK-2 inhibitor Neurofibromatosis type 1 EMA's Committee for Medicinal Products for Human Use recommended conditional marketing authorization to treat symptomatic, inoperable plexiform neurofibromas in children 3 and older
Astrazeneca plc, of Cambridge, U.K. Tagrisso (osimertinib) EGFR inhibitor Non-small-cell lung cancer  EMA's Committee for Medicinal Products for Human Use recommended marketing authorization to treat adults with early stage, EGFR-mutated disease after complete tumor resection
Axsome Therapeutics Inc., of New York AXS-05 (dextromethorphan + bupropion) NMDA receptor antagonist  Major depressive disorder FDA accepted NDA and granted priority review, setting PDUFA date of Aug. 22, 2021
Chugai Pharmaceutical Co. Ltd., of Tokyo Enspryng (satralizumab) Anti-IL-6 receptor monoclonal antibody Neuromyelitis optica spectrum disorder EMA's Committee for Medicinal Products for Human Use recommended marketing authorization to treat people 12 and older with anti-aquaporin-4 antibody seropositive disorder as monotherapy or in combination with immunosuppressive therapy 
Cstone Pharmaceuticals Co. Ltd., of Suzhou, China Gavreto (pralsetinib)  RET tyrosine kinase receptor inhibitor Thyroid cancer China's NMPA accepted NDA and designated priority review to treat advanced/metastatic RET-altered disease
Glenmark Pharmaceuticals Ltd., of Mumbai Ryaltris (olopatadine hydrochloride + mometasone furoate) Histamine H1 receptor antagonist Allergic rhinitis European Union approved fixed-dose combination nasal spray to treat people 12 and older
Johnson & Johnson, of New Brunswick, N.J. Ad26.COV2 (JNJ-78436735) SARS-CoV-2 recombinant adenoviral vector vaccine COVID-19 FDA and CDC allowed resumption of single-shot vaccine in people 18 and older under EUA
Karyopharm Therapeutics Inc., of Newton, Mass. Nexpovio (selinexor) Exportin 1 inhibitor Multiple myeloma EMA validated type II variation MAA to expand authorized indication to include, in combination with Velcade (bortezomib) and low-dose dexamethasone, adults who received at least 1 prior therapy; Committee for Medicinal Products for Human Use expected to complete its review in fourth quarter of 2021
Neoleukin Therapeutics Inc., of Seattle NL-201 Dual IL-15/IL-2 receptor agonist Solid tumors FDA removed clinical hold on IND application, allowing phase I trial to begin recruiting up to 120 people with advanced, relapsed or refractory disease
Scopus Biopharma Inc., of New York Immuno-oncology RNA therapy STAT3 gene inhibitor; TLR-9 agonist B-cell non-Hodgkin lymphoma IND for phase I trial submitted to FDA
Tarsier Pharma Ltd., of Tel Aviv, Israel TRS-02 Immunomodulator Back-of-eye blinding indications, including posterior/intermediate uveitis and panuveitis FDA provided positive feedback on development plan, including CMC requirements and bridging of non-clinical data, during pre-IND meeting
Uniqure NV, of Amsterdam Etranacogene dezaparvovec AAV gene therapy Hemophilia B FDA lifted clinical hold on pivotal Hope-B trial after determining issues related to single patient diagnosed with hepatocellular carcinoma were satisfactorily addressed; top-line 52-week data expected in second quarter of 2021
Vertex Pharmaceuticals Inc., of Boston, Mass., and Crispr Therapeutics AG, of Zug, Switzerland CTX-001 BCL11A gene modulator Beta-thalassemia EMA granted priority medicines designation to treat transfusion-dependent disease
Voyager Therapeutics Inc., of Cambridge, Mass. VY-HTT01 AAV gene therapy Huntington's disease FDA lifted clinical hold on phase I/II IND application; Vytal study expected to begin by year-end 2021

Notes

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