Drugs in the Pipeline

Lasmiditan NDA Submitted for Acute Treatment of Migraine

Lasmiditan NDA Submitted for Acute Treatment of Migraine

The NDA submission included data from two Phase 3 trials, SAMURAI and SPARTAN.

Glecaprevir/Pibrentasvir 8-Week Regimen Evaluated in Treatment-Naïve HCV Patients With Compensated Cirrhosis

Glecaprevir/Pibrentasvir 8-Week Regimen Evaluated in Treatment-Naïve HCV Patients With Compensated Cirrhosis

In EXPEDITION-8, 280 treatment-naïve HCV patients (genotypes 1, 2, 4, 5, and 6) with compensated cirrhosis were treated with Mavyret for 8-weeks; the primary efficacy measure was SVR12 rate.

Oral Therapy for Suicidal Bipolar Depression Gets Breakthrough Designation

Oral Therapy for Suicidal Bipolar Depression Gets Breakthrough Designation

NRX-101 combines D-cycloserine (DCS), an N-methyl-D-aspartate (NMDA) receptor antagonist, and lurasidone, a D2/5-HT2a receptor antagonist.

FDA Fast-Tracks Treatment for Pachyonychia Congenita

FDA Fast-Tracks Treatment for Pachyonychia Congenita

Through Palvella's proprietary QTORIN formulation, PTX-022 targets basal keratinocytes, which harbor the mutant keratin genes that cause PC.

FDA Grants Non-Opioid Analgesic VVZ-149 Fast Track Status

FDA Grants Non-Opioid Analgesic VVZ-149 Fast Track Status

The Company states that in vivo tests have shown that the compound's pain suppressing efficacy is comparable to morphine.

Cannabidiol Gets Orphan Drug Status for Preventing Ischemia-Reperfusion Injury

Cannabidiol Gets Orphan Drug Status for Preventing Ischemia-Reperfusion Injury

The designation was granted to Revive Therapeutics, a company that specializes in cannabis-based pharmaceuticals.

Nirogacestat Gets Fast-Tracked for the Treatment of Desmoid Tumors

Nirogacestat Gets Fast-Tracked for the Treatment of Desmoid Tumors

The Company is planning a Phase 3 study (DeFi) sometime in the first half of 2019.

FDA to Review Doptelet for Treatment of Chronic Immune Thrombocytopenia

FDA to Review Doptelet for Treatment of Chronic Immune Thrombocytopenia

Doptelet is a thrombopoietin receptor agonist (TPO-RA) that was approved in May 2018 to treat thrombocytopenia in adults with chronic liver disease who are scheduled to undergo a procedure.

Dupilumab Gets Priority Review for Adolescent Atopic Dermatitis

Dupilumab Gets Priority Review for Adolescent Atopic Dermatitis

Dupixent is also being assessed in other allergic/inflammatory conditions, including pediatric atopic dermatitis, pediatric asthma, chronic rhinosinusitis with nasal polyps, eosinophilic esophagitis, grass allergy, and peanut allergy.

NDA for Non-Opioid Combo Tx for Postoperative Pain Submitted to FDA

NDA for Non-Opioid Combo Tx for Postoperative Pain Submitted to FDA

The treatment is designed to deliver pain relief directly to the site of tissue injury, thereby potentially reducing the need for systemically administered pain medications.

Contepo NDA Submitted for Treatment of Complicated UTI

Contepo NDA Submitted for Treatment of Complicated UTI

Contepo, an investigational epoxide intravenous (IV) antibiotic, has broad spectrum activity against Gram-negative and Gram-positive pathogens, including multi-drug resistant strains such as ESBL-producing Enterobacteriaceae.

Dengue Vaccine Candidate Gets FDA's Priority Review

Dengue Vaccine Candidate Gets FDA's Priority Review

Two Phase 3 trials conducted in Asia and Latin America assessed the efficacy of the vaccine in >35,000 individuals aged 2 to 16 years.

Mitomycin Gel Granted Breakthrough Tx Designation for Urothelial Cancer

Mitomycin Gel Granted Breakthrough Tx Designation for Urothelial Cancer

UGN-1 is an investigational formulation of mitomycin, delivered to patients using standard intravesical catheters.

Development Program for Glut1 Deficiency Syndrome Drug to Be Discontinued

Development Program for Glut1 Deficiency Syndrome Drug to Be Discontinued

The decision was made after results from a Phase 3 trial (N=44) comparing UX007 to placebo did not show a statistically significant reduction in paroxysmal movement events in favor of the treatment (primary endpoint).

Jakafi Granted Priority Review for Acute Graft-Versus-Host Disease

Jakafi Granted Priority Review for Acute Graft-Versus-Host Disease

The sNDA included data from the REACH1 study (N=71) which evaluated ruxolitinib in combination with corticosteroids in patients with steroid-refractory acute GVHD.