The Genetic Rett Syndrome Drug from Acadia has Received FDA approval

The first medication Trofinetide to treat genetic Rett syndrome, a brain ailment, has been approved by the U.S. Food and Drug Administration, according to the manufacturer Acadia Pharmaceuticals. With a warning of diarrhea and weight loss, the U.S. health regulator’s decision permits the use of trofinetide, to be marketed as Daybue, in adult and pediatric patients two years of age and older. This approval comes months after the FDA rejected Acadia’s request to use Nuplazid for longer periods of time in the treatment of psychosis associated with Alzheimer’s disease.

According to analysts, Daybue’s approval would benefit the company’s growth in the short future. Before the approval, Acadia senior executive Kathie Bishop stated, “We have put a lot of thought into prospective commercialization of trofinetide, including resources for patients to get the medicine. By the end of April, Acadia claimed it would make the medication available to patients. It did not provide pricing information. Prior to the approval, David Hoang, an analyst at SMBC Nikko Securities, predicted that the list price at launch would be $450,000 per year. By 2035, he projected peak trofinetide sales in the US to be $487.2 million. Gregory Renza, an analyst at RBC Capital Markets, also penned a letter prior to clearance.

By 2032, peak U.S. sales are expected to surpass $500 million, with an annual average launch price of roughly $425,000. According to Refinitiv statistics, Acadia expects sales of Nuplazid, the only medication it has on the market, to be between $520 and $550 million this year, exceeding analysts’ consensus expectations of $532.8 million. Investors have placed their hopes in a successful trofinetide launch because the company would lose exclusivity for Nuplazid in 2028.

Acadia declared it will stop pursuing that usage of Nuplazid after the FDA rejected the drug’s extended use. The pharmaceutical company intends to concentrate its efforts on the early stages of ACP-204’s development for Alzheimer’s-related psychosis and the late stages of Nuplazid’s development to treat schizophrenia symptoms. Rett’s syndrome is an uncommon neurodevelopmental disorder that mostly affects females. Government estimates indicate that less than 50,000 Americans are affected. On Friday, shares of the California-based business decreased by 0.68 percent.

The medication from Acadia works as a synthetic version of the insulin-like growth factor IGF-1 and aids in the transmission of nerve impulses while reducing inflammation in the neurological tissue. Data from a late-stage study that indicated improvement in the disease’s primary symptoms after therapy with the medicine as opposed to a placebo served as the foundation for Acadia’s application for marketing authorisation. The Clinical Global Impression of Improvement and the Rett Syndrome Behaviour Questionnaire were used to assess the improvement in symptoms. Neuron Pharmaceuticals, an Australian pharmaceutical company, had granted the company a $10 million upfront license to develop and market the medicine in North America in 2018.

More about Genetic Rett Syndrome

Rett syndrome is a genetic disorder that affects the development of the brain, leading to severe intellectual disability, loss of language and motor skills, and various other physical and cognitive impairments. It is caused by mutations in the MECP2 gene, located on the X chromosome.

Rett syndrome almost exclusively affects females, as the mutation in the MECP2 gene is usually lethal in males. This is because males have only one X chromosome, and if that chromosome carries the mutation, they will not have a backup copy of the gene on a second X chromosome, unlike females.

The MECP2 gene codes for a protein called methyl-CpG-binding protein 2, which plays a critical role in regulating the expression of other genes. Mutations in the MECP2 gene disrupt this function, leading to abnormal brain development and the characteristic symptoms of Rett syndrome.

Although there is currently no cure for Rett syndrome, there are therapies and treatments available that can help manage the symptoms and improve the quality of life for those affected. Research into the underlying genetics and biology of Rett syndrome is ongoing, with the goal of developing more effective treatments and eventually a cure.

More about Acadia Pharmaceuticals

Acadia Pharmaceuticals is a biopharmaceutical company that develops and commercializes innovative medicines for the treatment of central nervous system disorders. The company was founded in 1993 and is headquartered in San Diego, California, USA.

Acadia Pharmaceuticals is primarily focused on developing therapies for neurological and psychiatric disorders, including Parkinson’s disease psychosis, schizophrenia, and Alzheimer’s disease. The company’s flagship product is pimavanserin, which is approved in the United States for the treatment of hallucinations and delusions associated with Parkinson’s disease psychosis.

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