Manuscript by da Costa, Anaelle; Metais, Thibaud; Mouthon, Franck; Kerkovich, Danielle; Charvériat, Mathieu, was successfully accepted by the journal “Fundamental & Clinical Pharmacology.”
The FDA has awarded Orphan Drug and Rare Pediatric Disease designations to Beyond Batten Disease Foundation for BBDF-101. The aim of the program is to facilitate the development of new drugs and biological products for the prevention and treatment of rare pediatric diseases. These designations provide accelerated review for approval, support with the FDA regulatory process and at least seven years of post-approval protection, as well as exemption from filing fees that normally have to be paid to the FDA. These designations do not impact the clinical trial, but are beneficial once the therapy receives New Drug Approval. We are grateful to Theranexus for their continued partnership and commitment to initiating a clinical trial for BBDF-101 as quickly as possible.
THERANEXUS AND BBDF OBTAIN ORPHAN DRUG DESIGNATION (ODD) AND RARE PEDIATRIC DISEASE DESIGNATION (RPDD) FROM THE FOOD AND DRUG ADMINISTRATION (FDA) FOR BBDF-101 FOR BATTEN DISEASE
Lyon, August 28, 2020 – Theranexus, a biopharmaceutical company innovating in the treatment of neurological diseases, and Beyond Batten Disease Foundation (BBDF) today announced the decision by the Food and Drug Administration (FDA) to award Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to the drug candidate BBDF-101 for Batten disease, a rare, fatal, genetic disorder of the nervous system for which there is no treatment.
The FDA has awarded Orphan Drug and Rare Pediatric Disease designations to Beyond Batten Disease Foundation for BBDF-101.
BBDF’s principal scientist, Danielle Kerkovich, recieves recognition for her paper as one of the most read in Molecular Genetics & Genomic Medicine.
Judy and Courtney Stecker establish the Charles “Wheeler” Stecker Research Fund to join forces in raising funds for a cure after their son Wheeler was diagnosed with Batten Disease.
Lyon, 12 December 2019 – Theranexus, a biopharmaceutical company innovating in the treatment of neurological diseases and pioneer in the development of drug candidates modulating the interaction between neurons and glial cells, is pleased to announce the signing of an agreement with Beyond Batten Disease Foundation, granting it a worldwide exclusive license to develop and commercialize the drug candidate BBDF-101 for juvenile Batten disease.
Bind Up Batten brought together the community on July 20, 2019, for the 2nd Annual Batten Disease Golf Charity Outing. Over 80 golfers came out on a beautiful, cloudy day in Waterville, Ohio to raise $12,700 for juvenile Batten Disease research.
In addition to the golfers, Bind Up Batten would like to thank the families, friends, musicians, Fallen Timbers Fairways and all the sponsors who provided their support to help make this event happen.
Special thanks and recognition to Mike’s parents TJ and John Klein as well as Donna and Paul Iwinski for their incredible effort along with many other volunteers who made this day possible.
Together we can Bind Up Batten!
We Are Austin did an interview highlighting Garland Benson and the Be a Hero Campaign
Check out his interview here:
If you had a goal to raise one million dollars in two years, you might think it would be impossible! Now imagine you’re 12 years old and setting out on this incredible mission. Well, Garland Benson did just that, and his perseverance will help fund research to treat his older sister’s degenerative disease. Garland is here this morning to talk about his remarkable success raising funds for Beyond Batten.
Thanks again to everyone who helped Garland hit his goal!
BBDF is actively pursuing a pharmaceutical partner to support the clinical trial and commercialize the treatment. Donations are rolling in and every additional dollar raised by Garland’s campaign will help offset activities required by the FDA to start the trial.
People Magazine has just published an article highlighting Garland’s quest to raise a million dollars for his sister.
Thanks again to everyone who helped Garland hit his goal!
BBDF is actively pursuing a pharmaceutical partner to support the clinical trial and commercialize the treatment. Donations are rolling in and every additional dollar raised by Garland’s campaign will help offset activities required by the FDA to start the trial.