Biologics

AskBio Gets Fast Track for Parkinson’s Gene Therapy

FDA has granted Bayer-owned Asklepios BioPharmaceutical (AskBio) a fast track designation for AB-1005, a gene therapy candidate for treating moderate Parkinson’s disease. The investigational adeno-associated virus 2 glial cell line-derived neurotrophic factor (AAV2-GDNF) neurorestorative gene therapy is designed for stable and continuous expression of GDNF in localized regions of the brain after direct neurosurgical injection with MRI-monitored convection enhanced delivery, according to the company.

“GDNF is a homodimer that is a distantly related member of the transforming growth factor-β superfamily,” AskBio explains. “In midbrain neuronal cell cultures, recombinant human GDNF promoted the survival and morphological differentiation of dopaminergic neurons and increased their high-affinity dopamine uptake. GDNF has long been evaluated as a potential disease modifying neurorestorative treatment for diseases, such as Parkinson’s, marked by progressive degeneration of midbrain dopaminergic neurons.”

Currently, the company says it is enrolling patients in the Phase 2 REGENERATE-PD clinical trial. In the randomized, double-blind, surgery-controlled trial, 87 subjects will receive either bilateral image-guided infusion of AB-1005 into the putamen, a single dose (active treatment arm), or bilateral partial burr/twist holes (control arm).

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome