FDA Approves First Oral Drug for Fabry Disease

FDA Approves First Oral Drug for Fabry Disease

The US Food and Drug Administration (FDA) has approved migalastat (Galafold, Amicus Therapeutics) for adults with Fabry disease and a genetic mutation determined to be amenable to treatment with the drug on the basis of in vitro assay data. Migalastat is the first oral medicine for Fabry disease and the first new therapy approved to […]

US Food And Drug Administration (FDA) Clears Natural Cycles As The First Digital Method Of Birth Control In The …

US Food And Drug Administration (FDA) Clears Natural Cycles As The First Digital Method Of Birth Control In The …

“We are aware of a significant number of women in the US looking for alternative, effective natural methods of birth control,” said Juan Acuna, M.D., Associate Professor of OBGYN, Department Chair at Florida International University College of Medicine. “Natural Cycles provides physicians with this much-needed natural and effective option to offer to appropriate patients.” The […]

Achromatopsia Treatment, AAV-CNGA3, Granted Orphan Drug Designation by FDA

Achromatopsia Treatment, AAV-CNGA3, Granted Orphan Drug Designation by FDA

This morning, August 13, 2018, the US Food and Drug Administration (FDA) has granted an orphan drug designation to MeiraGTx Holdings Plc’s AAV-CNGA3, a gene therapy, for the treatment of achromatopsia (ACHM) caused by mutations in the CNGA3 gene. “Without any currently approved therapies, we are very pleased by the FDA’s decision and the recognition from the […]

MeiraGTx (MGTX) Says AAV-CNGA3 Received Orphan Drug Designation from FDA for Treatment of Achromatopsia

MeiraGTx (MGTX) Says AAV-CNGA3 Received Orphan Drug Designation from FDA for Treatment of Achromatopsia

Get inside Wall Street with StreetInsider Premium. Claim your 2-week free trial here. MeiraGTx Holdings Plc (NASDAQ: MGTX), a vertically integrated, clinical stage gene therapy company, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) for its AAV-CNGA3 gene therapy product candidate for the treatment of achromatopsia (ACHM) […]

FDA Issues Draft Guidance on Measuring Efficacy of Medication-Assisted OUD Treatment

FDA Issues Draft Guidance on Measuring Efficacy of Medication-Assisted OUD Treatment

Agency encourages developers to consider other ways to evaluate patient outcomes when treating for opioid addiction. A PPM Brief In late May, FDA announced a challenge1 to the healthcare and device community to develop innovative technologies that would help could provide solutions for detecting, treating, and preventing opioid addiction, or Opioid Use Disorder (OUD). Now […]

First ever RNA-based gene-silencing drug approved by FDA, and it's not cheap

First ever RNA-based gene-silencing drug approved by FDA, and it's not cheap

Twenty years after the breakthrough discovery illustrating how RNA interference can be used to silence certain genes, and over a decade after the research won a Nobel prize, the U.S. Food and Drug Administration has approved the first drug utilizing this method for adult clinical treatment. The condition, hereditary transthyretin-mediated amyloidosis (hATTR), is rare and […]

Gottlieb: FDA will streamline drug safety evaluations

Gottlieb: FDA will streamline drug safety evaluations

T he Food and Drug Administration will soon standardize the way it handles data on the safety and effectiveness of drugs in an effort to reduce inconsistencies in the drug review process, agency Commissioner Scott Gottlieb said Friday. “Rather than just looking at drug safety parameters in terms of the tables that are submitted to us, […]

Alnylam's gene silencing drug wins FDA approval

Alnylam's gene silencing drug wins FDA approval

(Reuters) – Alnylam Pharmaceuticals Inc’s drug for a rare hereditary disease won U.S. regulatory approval on Friday, becoming the first approved treatment from a new class of medicines that use gene silencing technology. Alnylam’s patisiran, commercially named Onpattro, was approved to treat polyneuropathy in patients with hereditary ATTR amyloidosis, a potentially fatal condition that affects […]