Company Product Description Indication Status
Advicenne SA, of Paris Sibnayal (ADV-7103) Potassium citrate monohydrate + potassium bicarbonate  Distal renal tubular acidosis Approved by European Commission
Amicus Therapeutics Inc., of Philadelphia  AT-GAA (cipaglucosidase alfa co-administered with miglustat) Enzyme replacement therapy plus glucosylceramide synthase inhibitor Pompe disease Completed type B pre-BLA meeting with FDA and, based on final written communication from agency, company intends to complete rolling BLA submission for cipaglucosidase alfa and submit NDA for miglustat
Avrobio Inc., of Cambridge, Mass. AVR-RD-01 Lentiviral gene therapy Fabry disease Due to FDA recent conversion from accelerated to full approval of Fabrazyme (agalsidase beta, Sanofi SA), Avrobio can no longer pursue accelerated approval pathway with FAB-GT trial as currently designed and instead intends to discuss with FDA a registration trial with a primary efficacy endpoint of clearance of GL-3/Gb3 inclusions in biopsied renal peritubular capillaries as the basis for full approval
Astrazeneca plc, of Cambridge, U.K. Farxiga (dapagliflozin) SGLT2 inhibitor Chronic kidney disease FDA expanded approval of drug for use in reducing risk of sustained estimated glomerular filtration rate decline, end-stage kidney disease, cardiovascular death and hospitalization for heart failure in adults with CKD at risk of progression
Camurus AB, of Lund, Sweden Buvidal (buprenorphine) modified-release solutions for injection Opioid receptor mu modulator; ORL1 receptor agonist Opioid dependence Australia’s TGA approved label updates to include new higher monthly 160-mg dose; to allow for direct initiation to weekly product, removing requirement to be stabilized on sublingual buprenorphine prior to starting treatment with Buvidal; to include changes for contraindications in pregnancy and lactation to precautions
Chiesi Global Rare Diseases, a business unit of Parma, Italy-based Chiesi Farmaceutici SpA Ferriprox (deferiprone) Iron chelator Transfusional iron overload Approved by FDA for transfusional iron overload due to sickle disease or other anemias in adults and pediatric patients 3 and older
Clearside Biomedical Inc., of Alpharetta, Ga. Xipere (triamcinolone acetonide suprachoroidal injectable suspension) Glucocorticoid receptor agonist Macular edema associated with uveitis Resubmitted NDA to FDA, which includes response to complete response letter received Oct. 18, 2019
Galmed Pharmaceuticals Ltd., of Tel Aviv, Israel Aramchol Arachidyl amido cholanoic acid Nonalcoholic steatohepatitis and fibrosis China’s NMPA cleared IND for phase III Armor registrational study
Hutchmed Ltd., of Hong Kong and Shanghai Surufatinib Oral angio-immuno kinase inhibitor Pancreatic and extra-pancreatic neuroendocrine tumors Completed rolling NDA submission to FDA
Intra-Cellular Therapies Inc., of New York Lumateperone 5-HT2a receptor antagonist  Bipolar disorder FDA accepted for review supplemental NDA for use in treating depressive episodes associated with bipolar I or II disorder as monotherapy and as adjunctive therapy with lithium or valproate; agency set PDUFA date of Dec. 17, 2021
Jacobio Pharmaceuticals Ltd., of Beijing JAB-21822 KRAS G12C inhibitor Advanced solid tumors FDA cleared IND for trials to test in patients with advanced solid tumors with KRAS G12C mutation, including non-small-cell lung cancer and colorectal cancer; enrollment is set to start in second half of 2021
Moderna Inc., of Cambridge, Mass. COVID-19 vaccine COVID-19 spike glycoprotein modulator COVID-19 World Health Organization issued emergency use listing for prevention of COVID-19 in individuals 18 and older


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