Drugs in the Pipeline

Investigational Nebulized LAMA Shows Promise in COPD Trial

Investigational Nebulized LAMA Shows Promise in COPD Trial

Results found that rates of COPD exacerbations were lowest in the 175mcg treatment arm. Also, the adverse events and serious adverse events in the revefenacin group were comparable to the tiotropium group.

FDA Requests More Data for Unique Osteoporosis Drug Candidate

FDA Requests More Data for Unique Osteoporosis Drug Candidate

Dr. Pascale Richetta, head of bone and executive vice president, UCB, stated, "With all three pivotal romosozumab Phase 3 studies now included in the clinical evidence package, representing data from more than 11,000 patients, we are committed to bringing this important potential new treatment to those people living at risk of fragility fractures."

First-in-Class Drug for Primary Hyperoxaluria Gets Orphan Drug Designation

First-in-Class Drug for Primary Hyperoxaluria Gets Orphan Drug Designation

The Orphan Drug designation was supported by data from preliminary studies showing that ALLN-177 significantly decreased urinary and plasma oxalate in several rodent and porcine models.

FDA Fast Tracks New Oral Antifungal to Treat Valley Fever

FDA Fast Tracks New Oral Antifungal to Treat Valley Fever

VT-1598, an oral fungal CYP51 inhibitor, has shown high potency against pathogens such as molds, yeasts, and multi-drug resistant fungal pathogens (eg, Candida auris).

Cancer Peptide Vaccine Designated Orphan Drug for MDS

Cancer Peptide Vaccine Designated Orphan Drug for MDS

Data from a Phase 1/2 study in patients with MDS who had disease progression on or after first-line azacitidine treatment showed initial signs of clinical activity with DSP-7888, and that it was well tolerated in study patients.

Advanced Breast Cancer Drug Granted Priority Review

Advanced Breast Cancer Drug Granted Priority Review

The NDA included data from two trials, MONARCH 1 and MONARCH 2, which are a part of the comprehensive MONARCH clinical trial program.

Entrectinib Gains Orphan Drug Status for NTRK Fusion(+) Tumors

Entrectinib Gains Orphan Drug Status for NTRK Fusion(+) Tumors

Currently, entrectinib is being evaluated in the global, multicenter, open-label, potentially registration-enabling Phase 2 STARTRK-2 trial (Studies of Tumor Alterations Responsive to Targeting Receptor Kinases)

Trevyent NDA Submitted for Pulmonary Arterial Hypertension

Trevyent NDA Submitted for Pulmonary Arterial Hypertension

Trevyent contain treprostinil, a vasodilatory prostacyclin analogue. It works primarily through vasodilation of pulmonary and systemic arterial vascular beds, and inhibition of platelet aggregation.

SOBI003 Gains Orphan Drug Status for Rare Metabolic Disorder

SOBI003 Gains Orphan Drug Status for Rare Metabolic Disorder

According to the Company, SOBI003 is currently in the late pre-clinical phase and its first clinical trial is expected to commence in 2018.

Biomarin Submits BLA for New Phenylketonuria Therapy

Biomarin Submits BLA for New Phenylketonuria Therapy

The investigational drug is intended to substitute the deficient PAH enzyme with a PEGylated phenylalanine lyase enzyme to break down Phe.

Astellas Seeks Approval for Overactive Bladder Combo Therapy

Astellas Seeks Approval for Overactive Bladder Combo Therapy

"We look forward to FDA's review of our application for a potential new treatment option for the millions of people living with OAB," said Bernhardt Zeiher, MD, president of Development at Astellas.

Emicizumab Beneficial in Hemophilia A Patients With Inhibitors to FVIII

Emicizumab Beneficial in Hemophilia A Patients With Inhibitors to FVIII

The primary analysis of the study arm showed a statistically and clinically significant reduction in bleeding by 87% (risk rate [RR] 0.13; P<0.0001] with emicizumab prophylaxis.

Rituxan Biosimilar BLA Accepted for FDA Review

Rituxan Biosimilar BLA Accepted for FDA Review

The proposed indications are for the treatment of non-Hodgkin's lymphoma (NHL), chronic lymphocytic leukemia (CLL), rheumatoid arthritis (RA), granulomatosis with polyangiitis and microscopic polyangiitis.

New Antibiotic Designated QIDP for Two Indications

New Antibiotic Designated QIDP for Two Indications

The gel is hormone-free and can be used with other forms of contraception or used alone. The gel, supplied in a pre-filled applicator, can be self-applied.

NDA Resubmitted for Twirla Contraceptive Patch

NDA Resubmitted for Twirla Contraceptive Patch

In the NDA resubmission, the Company included safety and efficacy data from the Phase 3 trial SECURE, the manufacturing information, and a summary response to the CRL.