FDA: Approves New Therapy for Dravet Syndrome
The U.S. Food and Drug Administration approved Fintepla (fenfluramine), a Schedule IV controlled substance, for the treatment of seizures associated with Dravet syndrome in patients age 2 and older.
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FUSION HEALTHCARE SOLUTIONS: FUSION PPE Offers NIOSH and CDC Approved N95 Respirator Mask Produced by BYD
FUSION PPE Offers NIOSH and CDC Approved N95 Respirator Mask Produced by BYD, Backed by Warren Buffet, As Covid-19 Surges with Potential Second Wave, and FDA Reissues Emergency Use Authorizations (EUAs) Revising Which Respirators Can Be Decontaminated for Reuse
HEMOSHEAR THERAPEUTICS: Receives FDA Clearance of IND for Phase 2 Study of its Investigational Drug HST5040 for the Treatment of Methylmalonic Acidemia and Propionic Acidemia
HemoShear Therapeutics, a clinical stage company developing treatments for rare metabolic disorders, has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to conduct a phase 2 clinical trial of HST5040, an oral small molecule drug for the treatment of patients with methylmalonic acidemia (MMA) and propionic acidemia (PA).
ZOGENIX, INC.: FDA Approves FINTEPLA® (fenfluramine) for the Treatment of Seizures Associated with Dravet Syndrome
FINTEPLA® significantly and substantially reduced convulsive seizure frequency in patients whose seizures were not adequately controlled on other medications, as observed in two phase 3 placebo-controlled clinical trials
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FDA: Announces First of Its Kind Pilot Program to Communicate Patient Reported Outcomes from Cancer Clinical Trials
The U.S. Food and Drug Administration today launched Project Patient Voice, an initiative of the FDA’s Oncology Center of Excellence (OCE).
ABBVIE: FDA Accepts Supplemental Biologics License Application (sBLA) for BOTOX® (onabotulinumtoxinA) for the Treatment of Pediatric Patients with Neurogenic Detrusor Overactivity
Application Seeks to Extend Use of BOTOX® for Patients 5 to 17 Years Old
FDA: Approves First Therapy for Rare Disease that Causes Low Phosphate Blood Levels, Bone Softening
The U.S. Food and Drug Administration approved Crysvita (burosumab-twza) injection to treat patients age two and older with tumor-induced osteomalacia (TIO), a rare disease that is characterized by the development of tumors that cause weakened and softened bones. The tumors associated with TIO release a peptide hormone-like substance known as fibroblast growth factor 23 (FGF23) that lowers phosphate levels.
DICERNA: Receives Rare Pediatric Disease Designation From U.S. Food and Drug Administration for Nedosiran for Treatment of Primary Hyperoxaluria
Dicerna Pharmaceuticals, Inc. (Nasdaq: DRNA) (the “Company” or “Dicerna”), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today announced that the U.S. Food and Drug Administration (“FDA” or the “agency”) has granted rare pediatric disease designation for nedosiran, an investigational RNAi therapy being developed as a once-monthly treatment for primary hyperoxaluria (PH).
LOULOU FOUNDATION: CDKL5 Deficiency Disorder Voice of the Patient Report Submitted to FDA
The Loulou Foundation and the International Foundation for CDKL5 Research (IFCR) are proud to release the “CDKL5 Deficiency Disorder Voice of the Patient Report”, capturing the voice of patients living with this rare genetic disease that affects thousands of people.
EPIZYME: Announces U.S. FDA Accelerated Approval of TAZVERIK™ (tazemetostat) for Relapsed/Refractory Follicular Lymphoma
TAZVERIK Now Approved for Second Indication
ALMIRALL: Announces FDA Approval Of Updated Label For Seysara® (Sarecycline) Tablets
Seysara® is a novel oral antibiotic developed specifically for the treatment of acne
AETHLON: Announces FDA Approval of IDE Supplement for COVID-19 Patients
Allows for enrollment of up to 40 subjects in up to 20 centers
FRAGRANCE CREATORS: Applauds FDA for Continued Efforts to Promote Public Health
In a webinar for Fragrance Creators members, FDA delivered detailed instructions on registration and listing of hand sanitizers under the Agency's temporary guidance
PROTAGONIST THERAPEUTICS, INC.: Hepcidin Mimetic PTG-300 Receives U.S. FDA Orphan Drug Designation for the Treatment of Polycythemia Vera
Protagonist Therapeutics, Inc. (Nasdaq: PTGX) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for PTG-300 for the treatment of polycythemia vera.
FDA: Coronavirus (COVID-19) Update: FDA Issues Warning Letters to Companies Inappropriately Marketing Antibody Tests, Potentially Placing Public Health at Risk
Agency Continues Effort to Help Protect Public Health from Fraudulent Products
FDA: Coronavirus (COVID-19) Update: FDA Revokes Emergency Use Authorization for Chembio Antibody Test
The U.S. Food and Drug Administration revoked the emergency use authorization (EUA) of the Chembio Diagnostic System, Inc. (Chembio) DPP COVID-19 IgM/IgG System, a SARS-CoV-2 antibody test, due to performance concerns with the accuracy of the test.
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FDA: Approves First Treatment for Adult Onset Still’s Disease, a Severe and Rare Disease
The U.S. Food and Drug Administration approved Ilaris (canakinumab) injection for the treatment of Active Still’s disease, including Adult-Onset Still’s Disease (AOSD). Ilaris was previously approved for Systemic Juvenile Idiopathic Arthritis (SJIA) in patients aged 2 years and older.
GENEQUANTUM HEALTHCARE: World's first iLDC enabling ADC drug receives IND clearance by the US FDA
GeneQuantum Healthcare (GQ) has recently received the US IND approval for its in-house developed next generation antibody drug conjugate (ADC) asset, GQ1001.
MYLAN N.V.: Mylan and Biocon Announce U.S. FDA Approval of Semglee™ (insulin glargine injection)
FDA approval marks a significant milestone to help increase access and affordability of insulin for the millions of Americans living with diabetes
CURIS: Announces FDA Clearance of IND Application for CI-8993, the First-In-Class Monoclonal Anti-VISTA Antibody
Phase 1a/1b dose escalation study to begin in the second half of 2020