Company Product Description Indication Status
Betterlife Pharma Inc., of Vancouver, British Columbia AP-003 Inhalation-based interferon alpha 2b formula COVID-19 Meeting with FDA provided guidance for IND filing to start clinical trials in U.S.
Clinigen Group plc, of Burton Upon Kent, U.K. Totect Dexrazoxane hydrochloride Cardiomyopathy associated with doxorubicin FDA granted approval to broaden indication to include reducing incidence and severity of cardiomyopathy associated with doxorubicin administration in women with metastatic breast cancer who have received a cumulative doxorubicin dose of 300 mg/m² and who will continue to receive doxorubicin therapy to maintain tumor control
Eli Lilly and Co., of Indianapolis Bamlanivimab (LY-CoV555) Antibody  COVID-19 FDA granted emergency use authorization for treatment of mild to moderate COVID-19 in adults and pediatric patients, 12 and older, with a positive COVID-19 test, who are at risk for progressing to severe COVID-19 and/or hospitalization
Emmaus Life Sciences Inc., of Torrance, Calif. Endari  L-glutamine oral powder Sickle cell disease Submitted temporary license application to the National Health Regulatory Authority in the Kingdom of Bahrain
Forma Therapeutics Inc., of Watertown, Mass. FT-4202 Selective red blood cell pyruvate kinase-R activator Sickle cell disease European Commission granted orphan designation
Immune-Onc Therapeutics Inc., of Palo Alto, Calif. IO-202 Antagonist antibody Acute myeloid leukemia FDA granted orphan designation
Oryzon Genomics SA, of Madrid, Spain Vafidemstat Oral, CNS optimized LSD1 inhibitor Borderline personality disorder Received approval from Spanish Drug Agency (AEMPS) for CTA to start phase IIb trial
Praxis Precision Medicines Inc., of Cambridge, Mass. PRAX-114 GABA A receptor modulator Major depressive disorder Received a response from the FDA on its IND submission seeking to start a phase II/III trial, with the agency placing the IND on full clinical hold; Praxis expects to receive final comments from FDA within 30 days
Rafael Pharmaceuticals Inc., of Cranbury, N.J. CPI-613 (devimistat) Targets mitochondrial tricarboxylic acid cycle Metastatic pancreatic cancer FDA granted fast track designation
Supernus Pharmaceuticals Inc., of Rockville, Md. SPN-812 (viloxazine hydrochloride) Psychomodulator; norepinephrine uptake inhibitor Attention-deficit hyperactivity disorder FDA issued a complete response letter to NDA seeking approval for use in pediatric patients, ages 6-17; primary issue cited relates to company’s in-house laboratory that conducts analytical testing, which recently moved to a new location; no clinical safety or efficacy issues were identified 
Supernus Pharmaceuticals Inc., of Rockville, Md. SPN-830 (apomorphine infusion pump) Dopamine D2 receptor agonist Parkinson’s disease Received a refuse-to-file letter from FDA regarding NDA seeking approval for continuous treatment of motor fluctuations in Parkinson’s disease; agency determined the NDA was not sufficiently complete to permit a substantive review; company plans to seek guidance from FDA, including type A meeting

Notes

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