Drugs in the Pipeline

CPI-613 Gets Orphan Drug Designation for Peripheral T-Cell Lymphoma

CPI-613 Gets Orphan Drug Designation for Peripheral T-Cell Lymphoma

This first-in-class drug was previously granted Orphan Drug designation for the treatment of pancreatic cancer, acute myeloid leukemia, Burkitt lymphoma, and myelodysplastic syndromes.

DMD Treatment Gets Orphan Drug, Rare Pediatric Disease Designations

DMD Treatment Gets Orphan Drug, Rare Pediatric Disease Designations

DMD is predominately caused by out-of-frame deletions in the dystrophin gene, which results in absent or defective dystrophin protein, leading to progressive and irreversible muscle function loss.

FDA to Review Gimoti for Diabetic Gastroparesis in Adult Women

FDA to Review Gimoti for Diabetic Gastroparesis in Adult Women

The FDA has set a Prescription Drug User Fee Act target date of April 1, 2019.

Positive Results for Novel Osteoarthritis Pain Treatment in Phase 3 Trial

Positive Results for Novel Osteoarthritis Pain Treatment in Phase 3 Trial

Change in pain and physical function score from baseline were chosen as the co-primary endpoints.

Long-Acting 2-Drug Injectable Noninferior to Oral 3-Drug Combo in HIV Study

Long-Acting 2-Drug Injectable Noninferior to Oral 3-Drug Combo in HIV Study

The ATLAS study enrolled 570 patients infected with HIV-1; participants were randomized to either the experimental or active comparator arms.

Treatment for Bronchiolitis Obliterans Gains Orphan Drug Status

Treatment for Bronchiolitis Obliterans Gains Orphan Drug Status

Bronchiolitis obliterans is the leading cause of morbidity and mortality in the pulmonary transplant population with ≥50% of patients who receive a lung transplant developing the condition within 5 years.

Iclaprim Gets Priority Review for Acute Bacterial Skin/Skin Structure Infections

Iclaprim Gets Priority Review for Acute Bacterial Skin/Skin Structure Infections

Clinical data has indicated that the drug exhibits a low propensity for resistance development as well as a favorable tolerability profile.

FDA to Review Midazolam Nasal Spray for Seizure Clusters

FDA to Review Midazolam Nasal Spray for Seizure Clusters

The NDA includes data from the Phase 3 ARTEMIS 1 trial which evaluated the intranasal spray in 292 patients.

Elzonris Granted Priority Review for Blastic Plasmacytoid Dendritic Cell Neoplasm

Elzonris Granted Priority Review for Blastic Plasmacytoid Dendritic Cell Neoplasm

The Company announced results from a Phase 2 trial involving 45 patients with BPDCN earlier this year.

Xolair Designated Breakthrough Therapy for Food Allergies

Xolair Designated Breakthrough Therapy for Food Allergies

The FDA designation was supported by data from 7 studies evaluating the safety and efficacy of Xolair against various food allergens (eg, peanut, milk, egg, others).

Gene Therapy Gets Orphan Drug Designation for Severe Genetic Skin Disease

Gene Therapy Gets Orphan Drug Designation for Severe Genetic Skin Disease

Patients with TGM-1 deficiency may have chronic pronounced scaling of the skin with increased transepidermal water loss, as the TGM-1 enzyme is essential for facilitating the formation of the epidermal barrier, which prevents dehydration.

FDA Grants Orphan Drug Status to Angelman Syndrome Treatment

FDA Grants Orphan Drug Status to Angelman Syndrome Treatment

Delay or lack of speech, seizures, and walking/balance disorders are some of the symptoms associated with AS.

Vicinium Fast-Tracked for Non-Muscle Invasive Bladder Cancer

Vicinium Fast-Tracked for Non-Muscle Invasive Bladder Cancer

A Phase 3 trial (Vista) of the treatment is currently taking place; the trial is evaluating the treatment in patients with NMIBC who have previously received 2 courses of BCG and who are now BCG-unresponsive.

Omadacycline Gets FDA Committee Nod for Skin Infections, Pneumonia

Omadacycline Gets FDA Committee Nod for Skin Infections, Pneumonia

The Committee's recommendation was based on data from the omadacycline global development program that included nearly 2000 adults in three Phase 3 studies.

Monoclonal Antibody Granted Orphan Drug Status for Frontotemporal Dementia

Monoclonal Antibody Granted Orphan Drug Status for Frontotemporal Dementia

There are currently no treatments available for frontotemporal dementia.