Under the EMA's Regulation (EC) No. 141/2000 an orphan medicinal product designation gives companies access to reduced regulatory fees, protocol assistance and guidance on preparing a dossier that will meet European regulatory requirements and thereby maximize the chance of success at the time of marketing authorization. Once approved, an orphan drug is also granted 10 years of market exclusivity in the European Union (EU), hence protecting it from competition from similar medicines, which cannot be marketed during this 10-year exclusivity period for this indication.
The EMA grants orphan medicinal product designation based upon several criteria: the life threatening and debilitating nature of the condition; the medical plausibility of the proposed orphan indication; a prevalence in Europe of less than 5 cases for each 10,000 of population; no satisfactory method of diagnosis, prevention or treatment exists or if such method exists the medicinal product will be of significant benefit to those affected by that condition.
ANAVEX®2-73 had previously received orphan drug designation from the United States (U.S.) Food and Drug Administration (FDA) for the treatment of Rett syndrome.
"ANAVEX®2-73 has the potential to provide patients and physicians with a much-needed treatment option for Rett syndrome, a rare genetic disorder. The COMP's adoption of a positive opinion for ANAVEX®2-73 orphan drug designation is another important milestone for this program, which continues to advance rapidly," said Christopher U Missling, PhD, President and Chief Executive Officer of Anavex. "In addition to Rett syndrome1, Anavex has ongoing clinical development programs for ANAVEX®2-73 for the treatment of Alzheimer's disease2 and Parkinson's disease dementia3."
About Rett Syndrome
Rett syndrome is a rare, non-inherited genetic postnatal progressive neurodevelopmental disorder that occurs almost exclusively in girls and leads to severe impairments, affecting nearly every aspect of the child's life: their ability to speak, walk, eat and even breathe easily. The hallmark of Rett syndrome is near constant repetitive hand movements while awake. It is characterized by normal early growth and development (6 to 18 months) followed by a slowing of development, loss of purposeful use of the hands, distinctive hand movements, slowed brain and head growth, problems with walking, seizures and intellectual disability. There is currently no cure for Rett syndrome and treatment of the disorder is symptomatic. Management of symptoms is done through a multidisciplinary approach utilizing medication for motor difficulties, breathing irregularities and control of seizures through anticonvulsant drugs. Rett syndrome is caused by mutations in the MECP2 gene and strikes all racial and ethnic groups and occurs worldwide in approximately one in every 10,000 to 15,000 live female births.
ANAVEX®2-73 activates the Sigma-1 receptor (S1R) protein, which serves as a molecular chaperone and functional modulator involved in restoring homeostasis. In a Phase 2a Alzheimer's disease (AD) study, ANAVEX®2-73 has shown dose dependent improvement in exploratory endpoints of cognition (MMSE) and activities of daily living (ADCS-ADL). Full genomic analysis of ANAVEX®2-73 Phase 2a AD patients was performed. The ANAVEX®2-73 Phase 2 Rett syndrome study design includes genomic biomarkers identified in the ANAVEX®2-73 Phase 2a AD study. Studies of ANAVEX®2-73 in a mouse model with a heterozygous Mecp2-null mutation (HET) that causes neurological symptoms that mimic Rett syndrome, ANAXEX®2-73 was evaluated in automatic visual responses and breathing tests in 7-month old mice, an age at which advanced pathology is evident. Vehicle-treated HET mice demonstrated fewer automatic visual responses and more frequent expiratory apneas than wild-type mice. Treatment with ANAVEX®2-73 for four weeks significantly increased these visual responses in the HET mice (p<0.05). Additionally, chronic oral dosing daily for 3-6.5 weeks of ANAVEX®2-73 starting at ~5 weeks of age was also conducted in the HET mouse model of Rett syndrome, and dose-dependent improvements in a variety of sensory and motor deficits, including those involving motor coordination, balance, and learning, were also observed. Notably, one of the strongest effects was on hindlimb clasping, a postural response that resembles the characteristic hand stereotypies present in Rett syndrome. These experiments were sponsored by Rettsyndrome.org.
About Anavex Life Sciences Corp.
Anavex Life Sciences Corp. (Nasdaq: AVXL) is a publicly traded biopharmaceutical company dedicated to the development of differentiated therapeutics for the treatment of neurodegenerative and neurodevelopmental diseases including Alzheimer's disease, Parkinson's disease, Rett syndrome and other central nervous system (CNS) diseases, pain and various types of cancer. Anavex's lead drug candidate, ANAVEX®2-73, recently completed a successful Phase 2a clinical trial for Alzheimer's disease. ANAVEX®2-73 is an orally available drug candidate that restores cellular homeostasis by targeting sigma-1 and muscarinic receptors. Preclinical studies demonstrated its potential to halt and/or reverse the course of Alzheimer's disease. ANAVEX®2-73 also exhibited anticonvulsant, anti-amnesic, neuroprotective and anti-depressant properties in animal models, indicating its potential to treat additional CNS disorders, including epilepsy. The Michael J. Fox Foundation for Parkinson's Research previously awarded Anavex a research grant, which fully funded a preclinical study to develop ANAVEX®2-73 for the treatment of Parkinson's disease. ANAVEX®3-71, which targets sigma-1 and M1 muscarinic receptors, is a promising preclinical drug candidate demonstrating disease-modifying activity against the major hallmarks of Alzheimer's disease in transgenic (3xTg-AD) mice, including cognitive deficits, amyloid and tau pathologies. In preclinical trials, ANAVEX®3-71 has shown beneficial effects on neuroinflammation and mitochondrial dysfunction. Further information is available at www.anavex.com. You can also connect with the company on Twitter,Facebook and LinkedIn.
Statements in this press release that are not strictly historical in nature are forward-looking statements. These statements are only predictions based on current information and expectations and involve a number of risks and uncertainties. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks set forth in the Company's most recent Annual Report on Form 10-K filed with the SEC. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Anavex Life Sciences Corp. undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof.
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1 ClinicalTrials.gov Identifier: NCT03758924; NCT03941444
2 ClinicalTrials.gov Identifier: NCT03790709
3 ClinicalTrials.gov Identifier: NCT03774459
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