Orphan-Drug Designation for Davunetide.
A Historic Moment in ADNP Syndrome History!
A Historic Moment in ADNP Syndrome History!
February 25, 2018
The ADNP Kids Research Foundation is excited to announce that Coronis Neurosciences has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for CP201 (davunetide). This marks the first ever possible treatment for patients with ADNP syndrome.
The ADNP Kids Research Foundation is excited to announce that Coronis Neurosciences has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for CP201 (davunetide). This marks the first ever possible treatment for patients with ADNP syndrome.
ADNP Syndrome, also known as Helsmoortel-VanDerAa Syndrome, is a rare neurodevelopmental disorder caused by a mutation in the ADNP (Activity Dependent Neuroprotective Protein) gene. This gene affects brain formation and development, as well as brain function. In addition to the brain, the ADNP gene also affects many other organs and functions of the body. It can cause the neurological, cardiovascular, endocrine, immune, musculoskeletal and gastrointestinal systems, as well as vision, hearing, growth, feeding and sleep. It can also cause mild to severe delays in intelligence, speech and motor planning, (including gross motor, fine motor and oral motor). It causes behavior disorders such as Autism Spectrum Disorder (ASD) in a substantial proportion of cases. In fact, it is estimated: ADNP to be mutated in at least 0.17% of genetic autism cases, making it one of the most frequent ASD-associated genes known to date.
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Devunetide is derived from a naturally occurring neuroprotective brain protein known as activity-dependent neuroprotective protein (ADNP). CP201 (devunetide) is a small fragment of Activity Dependent Neuroprotective Protein (ADNP); it is an important nerve cell protecting snippet that enhances ADNP activity. It has been extensively studied in more than 20 cell assays and 25 animal models for autism, neuropsychiatric and neurodegenerative diseases. The developers websites claims that CP201 improves learning and memory, increases nerve cell plasticity, protects the cell scaffold and transport system, and restores the autophagic function. The full description can be found on coronisns.com.
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“This is a major achievement for Coronis which secures the US market for CP201 for the treatment of ADNP syndrome, one of the three most common genetically dependent neurodevelopmental impairments within the Autism spectrum.” Dr. Eric Messika, President and CEO of Coronis. |
Dr. Eric Messika, President and CEO of Coronis, said “this is a major achievement for Coronis which secures the US market for CP201 for the treatment of ADNP syndrome, one of the three most common genetically dependent neurodevelopmental impairments within the Autism spectrum.”
“This marks a significant step on the road to effective approved treatments for ADNP,” said Sandra Sermone, ADNP-KRF President. “Patients with ADNP syndrome currently do not have one single FDA approved drug for treatment of ADNP syndrome. The ADNP Kids Research Foundation is proud to be working with the team at Coronis on this monumental novel treatment project.”
Based on this granted status Coronis is now planning the US portion of their upcoming ADNP clinical trial. The group plans to perform a prospective observational study in a small group of ADNP children to generate descriptive statistics and inform assumptions for several end-point assessments planned to be used in a subsequent pivotal interventional study. These studies will be performed the Seaver Autism Center at Mount Sinai Hospital in New York City, Directed by Prof. Joseph Buxbaum and the Autism Center at the Seattle Hospital in Washington, directed by Prof. Raphael Bernier.
If you are interested in information and future participation on this and other studies, please fill out the contact information form on our RESEARCH page.
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