Drugs in the Pipeline

Investigational Migraine Tx Shows Promise in Phase 3 Trial

Investigational Migraine Tx Shows Promise in Phase 3 Trial

Results demonstrated that at 12 weeks after the first dose of fremanezumab, the CM group had reductions of monthly headaches; -4.6 days for the monthly dose, -4.3 days for the quarterly dose and -2.5 for the placebo group (P<0.0001).

Cabometyx sNDA Submitted for Expanded Renal Cell Carcinoma Indication

Cabometyx sNDA Submitted for Expanded Renal Cell Carcinoma Indication

"If [the sNDA is] approved, Cabometyx will offer an important new alternative for the treatment of patients with previously untreated advanced RCC," said Michael M. Morrissey, PhD, President and CEO of Exelixis.

Romosozumab Followed by Alendronate Superior in Reducing Fracture Risk

Romosozumab Followed by Alendronate Superior in Reducing Fracture Risk

ARCH (Active-contRolled FraCture Study in Postmenopausal Women with Osteoporosis at High Risk of Fracture) was a multicenter, international, randomized, double-blind, alendronate-controlled study (n=4,093) in postmenopausal women with osteoporosis at high risk for fracture based on previous fracture history.

Dupilumab Beneficial in Patients with Uncontrolled, Persistent Asthma

Dupilumab Beneficial in Patients with Uncontrolled, Persistent Asthma

Results found that at 52 weeks severe asthma attacks were reduced by 46% in the overall population for the 300mg dose group and by 60% and 67% among those patients with ≥150 eosinophilic cells/microliter or greater and ≥300 eosinophilic cells/microliter, respectively (P<0.001).

Cemiplimab Gains Breakthrough Tx Designation for Advanced Skin Cancer

Cemiplimab Gains Breakthrough Tx Designation for Advanced Skin Cancer

A Phase 2, single-arm, open-label clinical trial, EMPOWER-CSCC 1, is now enrolling patients with metastatic CSCC and locally advanced and unresectable CSCC.

Pediatric Ulcerative Colitis Tx Granted Orphan Drug Designation

Pediatric Ulcerative Colitis Tx Granted Orphan Drug Designation

Early Phase 1 data has indicated that the treatment drug can be more efficacious than mesalamine/5-ASA treatments alone.

Oral Tx for Acute Suicidal Ideation and Behavior Gets Fast Track Status

Oral Tx for Acute Suicidal Ideation and Behavior Gets Fast Track Status

Results from two previously Phase 2 trials of NeuroRx's sequential treatment have shown a 50% reduction in symptoms of depression and a 75% reduction in suicidal ideation in bipolar patients.

Elagolix NDA Submitted for Treating Endometriosis-Associated Pain

Elagolix NDA Submitted for Treating Endometriosis-Associated Pain

Elagolix, an oral gonadotropin-releasing hormone (GnRH) antagonist, was evaluated in two Phase 3 clinical studies in about 1,700 women with moderate-to-severe endometriosis-associated pain.

Familial Chylomicronemia Syndrome Treatment to Get FDA Review

Familial Chylomicronemia Syndrome Treatment to Get FDA Review

A total of 66 patients with FCS were enrolled in the year-long APPROACH which met its primary endpoint of reduction in triglycerides at 3 months, with a 77% mean reduction in triglycerides for volanesorsen-treated patients.

FDA Greenlights MDMA Studies for Posttraumatic Stress Disorder

FDA Greenlights MDMA Studies for Posttraumatic Stress Disorder

"Our Phase 2 data was extremely promising with a large effect size, and we are ready to move forward quickly," said Amy Emerson, Executive Director of the MAPS Public Benefit Corporation.

FDA to Review Novel ADHD Treatment Dasotraline

FDA to Review Novel ADHD Treatment Dasotraline

Two year-long studies assessing safety of dasotraline in children, adolescent and adult ADHD patients found the treatment to be generally well tolerated.

Tezacaftor/Ivacaftor Gets Priority Review for Cystic Fibrosis

Tezacaftor/Ivacaftor Gets Priority Review for Cystic Fibrosis

The FDA has granted Priority Review to the New Drug Application (NDA) of tezacaftor/ivacaftor (Vertex) for the treatment of patients ≥12yrs old with cystic fibrosis (CF) who have two copies of the F508del mutation or one F508del mutation and one residual function mutation.

BLA for Novel Hemophilia A Therapy Granted Priority Review

BLA for Novel Hemophilia A Therapy Granted Priority Review

The FDA has accepted and granted Priority Review to the Biologics License Application (BLA) of emicizumab (Genentech) for the prophylactic treatment of hemophilia A in adults, adolescents and children with factor VIII inhibitors.

BLA Submitted for Novel X-Linked Hypophosphatemia Tx

BLA Submitted for Novel X-Linked Hypophosphatemia Tx

Burosumab is being developed in a collaboration between Ultragenyx and Kyowa Hakko Kirin Pharmaceuticals, based on a license agreement between both companies.

Adcetris sBLA Granted Priority Review for Cutaneous T-Cell Lymphoma

Adcetris sBLA Granted Priority Review for Cutaneous T-Cell Lymphoma

Key secondary endpoints included complete response rate, progression-free survival (PFS), and reduction in the burden of symptoms during treatment.