RDR Alert®

A review of novel drug approvals in 2018 found more than half of all novel approvals were for rare diseases and that 2018 also saw more novel drug approvals for rare disease than any other year.

Dawn Rotellini, chief operating officer of the National Hemophilia Foundation, discusses the important of including patient advocacy groups in annual meetings such as the NORD Summit.

A new analysis of an open-label extension study of the phase 3 PREVENT study is providing further evidence of the safety and efficacy of the treatment of the neuromyelitis optica spectrum disorder.

A new study presented at NORD Summit 2019 found rare disease is underrepresented in published literature and, more often than not, articles are not available in open-access journals.

Moira Gunn, PhD, professor of bioentrepreneurship at the University of San Francisco, discusses why it is important for primary care providers to keep up-to-date on rare disease developments.

Dawn Rotellini, chief operating officer of the National Hemophilia Foundation, discusses the impact recent advances in technology and changes in policy focusing on rare diseases have impacted the disease state of hemophilia.

Lucas Kempf, MD, associate director of the FDA's Rare Diseases Program, discusses the impact of recent policy changes that center around increasing access to and creating new therapies for rare disease.

The FDA is allowing Rocket Pharmaceuticals to conduct a global phase I study testing a new treatment approach for Pyruvate Kinase Deficiency.

The newly approved drug is a combination of 3 drugs that target the most common cystic fibrosis mutation.

The indication expands the therapy to include newly diagnosed and persistent adults ITP patients who have had an insufficient response to therapy.

The US Food and Drug Administration has approved minocycline topical foam, 4% for the treatment of inflammatory lesions of non-nodular moderate to severe acne vulgarism in adults and pediatrics patients 9 years of age or older.

Investigative drug DB102 will be assessed in approximately 200 patients with newly diagnosed glioblastoma in a Denovo-led phase 2b trial.

The new implanted drug will help treat adults suffering from erythropoietic protoporphyria with a history of photoxic reactions.

Gail Rodich, MD, discusses what the approval of rituximab, which has approvals for 9 other conditions including rheumatoid arthritis, means for pediatric patients with GPA and MPA.

The indication for the add-on monoclonal antibody makes it the first approved therapy for children with the rare vasculitis conditions.

The approval is based on findings showing patients on the drug plus VTd had a 53% reduction in risk of disease progression or death, compared to lone VTd.

The approval for interstitial lung disease associated with systemic sclerosis or scleroderma makes nintedanib the first therapy for the rare condition.

The once-daily capsule therapy is the first drug indicated for the rare disease since ruxolitinib in 2011.

The investigative monoclonal antibody from Regeneron shows significant benefit for patients with the rare, severe homozygous familial hypercholesterolemia.

The FDA has been informed by the therapy's manufacturer that data was manipulated during its animal testing phase.

AbbVie's submitted NDA for elagolix for the treatment of heavy menstrual bleeding associated with uterine fibroids in women is supported by the results of 2 phase 3 studies.

The therapy, which carries a Boxed Warning for liver injury risks, is indicated for patients who do not benefit from surgery.

Investigators find improved liver function in hepatocellular carcinoma patients tied to aspirin use in a new retrospective review.

FDA announces 137 new drug approvals in 2018 represents an 11% increase over 2017.

The recommendation was based on the SENSCIS trial results, orginally shared at ATS 2019 this year.

The selective inhibitor was previously approved to treat plaque psoriasis and psoriatic arthritis.

Merck announced the FDA's approval of their NDA for imipenem, cilastatin, and relebactam (Recarbrio) on Wednesday morning.

Former Vice President Joe Biden's presidential campaign has resulted in the nonprofit organization's indefinite suspension.

The FDA announced the approval of the nuclear export inhibitor based on the results of the STORM study and the ongoing BOSTON study.

The CD38 monoclonal antibody has been approved in combination with lenalidomide and dexamethasone to treat patients ineligible for autologous stem cell transplant.