A Potential Methylmalonic Acidemia Therapy Will be Developed Under the FDA’s START Pilot Program
source: shutterstock.com

A Potential Methylmalonic Acidemia Therapy Will be Developed Under the FDA’s START Pilot Program

Rare diseases are challenging. Not just for those affected, but for researchers, physicians, and drug developers. Rare diseases may affect small populations. Many are poorly understood and lack adequate awareness.…

Continue Reading A Potential Methylmalonic Acidemia Therapy Will be Developed Under the FDA’s START Pilot Program
New FDA Regulation on Laboratory Developed Tests Draws Objections from Healthcare Groups
source: shutterstock.com

New FDA Regulation on Laboratory Developed Tests Draws Objections from Healthcare Groups

As reported in Biopharma Dive, the FDA’s decision to strengthen its authority involving laboratory developed tests (LDTs) has drawn considerable objections from various healthcare groups. However, the Agency has held…

Continue Reading New FDA Regulation on Laboratory Developed Tests Draws Objections from Healthcare Groups

Barth Syndrome Chairman Appeals to the FDA For Appropriate Guidelines When Approving Drugs for Ultra-Rare Diseases

According to an article submitted to StatNews by members of the Barth Syndrome Foundation, the FDA approved the drug Aduhelm in accordance with its accelerated approval program. Although the treatment…

Continue Reading Barth Syndrome Chairman Appeals to the FDA For Appropriate Guidelines When Approving Drugs for Ultra-Rare Diseases
Second-Ever Therapy for Waldenström’s Macroglobulinemia is Now FDA Approved
source: pixabay.com

Second-Ever Therapy for Waldenström’s Macroglobulinemia is Now FDA Approved

The FDA has just announced their approval of zanubrutinib (Brukinsa) as a therapeutic option for Waldenström's macroglobulinemia. This approval was given based on the ASPEN Phase 3 clinical trial, and…

Continue Reading Second-Ever Therapy for Waldenström’s Macroglobulinemia is Now FDA Approved
FDA Clears IND Application For NT-I7, a PML Treatment
source: pixabay.com

FDA Clears IND Application For NT-I7, a PML Treatment

The FDA has recently accepted NeoImmuneTech's Investigational New Drug (IND) application for NT-17, a treatment for progressive multifocal leukoencephalopathy (PML). Now that the application has been cleared, the biopharmaceutical company…

Continue Reading FDA Clears IND Application For NT-I7, a PML Treatment
CRN04777 Given Rare Pediatric Disease Designation for Congenital Hyperinsulinism
source: pixabay.com

CRN04777 Given Rare Pediatric Disease Designation for Congenital Hyperinsulinism

  In a recent press release, clinical-stage pharmaceutical company Crinetics Pharmaceuticals announced that their investigational drug candidate, CRN04777, received Rare Pediatric Disease designation from the FDA. The therapy is designed…

Continue Reading CRN04777 Given Rare Pediatric Disease Designation for Congenital Hyperinsulinism