Therapeutic Specific Areas

FDA leaders insist that the decision to approve Sarepta’s Exondys (eteplirsen) should not be considered a model for future development of orphan drugs.

The agency awarded 21 new clinical trial research grants to boost the development of treatments for rare diseases.

The agency granted accelerated approval to Sarepta’s controversial DMD treatment, under the condition the company conduct additional studies to demonstrate efficacy.

The cyclin dependent kinase inhibitor received Breakthrough Therapy Designation as a first line treatment for advanced breast cancer.

The mAb, in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, was granted a Breakthrough Therapy Designation from FDA for the treatment of multiple myeloma.

The agency says the increasing requests for orphan drug designation has resulted in a change in FDA’s review goals.

The drug received breakthrough therapy and orphan drug designation as a monotherapy for the treatment of chronic graft-versus-host-disease.

Emergent is seeking approval for the manufacture of BioThrax at the company’s large-scale manufacturing facility.

The agency published guidance on the nonclinical evaluation of osteoporosis treatments.

FDA approved Vaxchora intended for travelers who are at risk for the disease.

Cinqair is approved for patients that have a history of severe asthma attacks despite receiving their current asthma medication.

The program is intended to provide support to ongoing efforts in rare disease product development.

FDA granted Immunomedics breakthrough therapy designation for the company’s investigational antibody drug conjugate for treatment of triple negative breast cancer.

FDA and industry seek a more consistent, flexible CMC review process for breakthrough therapies.

Health Canada approved AbbVie’s Humira for the treatment of adults with hidradenitis suppurativa.

FDA releases a report that analyses why some diseases are lacking treatment options.

The agency issues draft guidance on the development of drugs to treat Duchenne muscular dystrophy.

Boehringer Ingelheim announces FDA approval of Stiolto Respimat inhaler for the treatment of COPD.

Dicerna Pharmaceuticals announces that FDA granted its primary hyperoxaluria type 1 (PH1) treatment Orphan Drug designation.

Pfizer's Trumenba is the first FDA-approved immunization against meningococcal disease for individuals 10-25 years old.

European Medicines Agency clarifies advanced-therapy medicinal products classification.