Drugs in the Pipeline

Pexidartinib Gets Priority Review for Tenosynovial Giant Cell Tumor

Pexidartinib Gets Priority Review for Tenosynovial Giant Cell Tumor

The NDA is supported by data from the Phase 3 ENLIVEN study (N=120) which randomized patients with symptomatic TGCT to receive pexidartinib or placebo to evaluate the safety and efficacy of the treatment.

FDA to Review Two Antibacterial Agents for Potentially Life-Threatening Infections

FDA to Review Two Antibacterial Agents for Potentially Life-Threatening Infections

Both filings have been granted Priority Review by the Agency.

Novel Glioblastoma Treatment Granted Orphan Drug Designation

Novel Glioblastoma Treatment Granted Orphan Drug Designation

In preclinical models, the inhibition of phosphorylated STAT3 (p-STAT3) by WP1066 resulted in the direct attack of various tumor cells.

FDA to Review Cenobamate for the Treatment of Partial-Onset Seizures

FDA to Review Cenobamate for the Treatment of Partial-Onset Seizures

While the exact mechanism of action of cenobamate is not fully understood, it is believed to work through a dual mechanism.

Baricitinib Beneficial in Two Phase 3 Atopic Dermatitis Trials

Baricitinib Beneficial in Two Phase 3 Atopic Dermatitis Trials

Baricitinib, a Janus kinase inhibitor, is currently approved under the brand name Olumiant for the treatment of moderately-to-severely active rheumatoid arthritis.

FDA Rejects Opioid System Modulator NDA for Depression Treatment

FDA Rejects Opioid System Modulator NDA for Depression Treatment

Alkermes plans to meet with the FDA to discuss next steps for ALKs 5461.

Investigational Treatment for Multiple System Atrophy Gets Orphan Drug Status

Investigational Treatment for Multiple System Atrophy Gets Orphan Drug Status

Currently, there are no approved therapies for the treatment of MSA, a rare progressive neurodegenerative disease.

Trials Evaluating Crenezumab in Early Alzheimer Disease Halted

Trials Evaluating Crenezumab in Early Alzheimer Disease Halted

Crenezumab is an investigational monoclonal antibody that preferentially binds to and promotes the removal of neurotoxic oligomers, a form of beta-amyloid.

Novel Combined Oral Contraceptive Effective in Phase 3 Trial

Novel Combined Oral Contraceptive Effective in Phase 3 Trial

Previous Phase 2 studies confirmed esterol has minimal impact on liver cells and metabolic pathways, as well as on coagulation parameters.

FDA Grants Breakthrough Therapy to 15-Valent Pneumococcal Conjugate Vaccine

FDA Grants Breakthrough Therapy to 15-Valent Pneumococcal Conjugate Vaccine

Phase 3 studies investigating safety, tolerability, and immunogenicity in both adults and children are currently underway.

Tanezumab Beneficial in Second Phase 3 Osteoarthritis Pain Trial

Tanezumab Beneficial in Second Phase 3 Osteoarthritis Pain Trial

Tanezumab is a humanized monoclonal antibody that selectively targets, binds to, and inhibits nerve growth factor (NGF).

Dengue Vaccine Candidate TAK-003 Looks Promising in Phase 3 Trial

Dengue Vaccine Candidate TAK-003 Looks Promising in Phase 3 Trial

The TIDES trial is investigating the safety and efficacy of 2 doses of TAK-003 in preventing dengue fever caused by any of the 4 dengue virus serotypes.

Bermekimab Looks Promising for Hidradenitis Suppurativa

Bermekimab Looks Promising for Hidradenitis Suppurativa

This investigational treatment was evaluated in 42 HS patients, of which 24 had previously failed anti-tumor necrosis factor (TNF) therapy.

FDA to Review Pifeltro, Delstrigo for Expanded HIV Indication

FDA to Review Pifeltro, Delstrigo for Expanded HIV Indication

Once-daily Pifeltro and Delstrigo were initially approved in August 2018 to treat HIV-1 infection in adults with no prior antiretroviral treatment experience.

First-in-Class Drug for Biliary Atresia Granted Orphan Drug Designation

First-in-Class Drug for Biliary Atresia Granted Orphan Drug Designation

Currently, there is no FDA-approved treatment for biliary atresia, a rare pediatric liver disease.