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Therapeutic Amicus, the global biotech company targeting therapies for rare diseases, announced that it the discontinuation of its gene therapy program for CLN6 Batten disease after studies failed to yield consistent results.

The program was unable to stabilize the disease control program for its two years, the company disclosed in a statement. The company is currently working to assess and share the phase I/II data it has collected so far with key stakeholders in the CLN6 Batten disease community, with the goal of generating support for find better ways of treating a disease that has a significant impact. unmet needs.

CLN6 disease is a disorder called neuronal ceroid lipofuscinoses (NCL), which has symptoms such as epilepsy, vision loss, speech problems, cognitive problems, and poor coordination and balance. Two to four out of every 100,000 children are diagnosed with the disorder, which usually begins between the ages of 5 and 8. For now, the only United States Food and drug administration– The approved treatment for Batten disease is cerliponase alfa, an enzyme replacement therapy that slows the progression of gait and coordination symptoms.

Although Amicus has decided to discontinue its CLN6 program, the company said it is advancing its CLN3 Batten disease program, this time with a different promoter, higher dose, and intra-cisterna magna route of administration. (ICM). The next steps in this effort will depend on the delivery of Phase I/II clinical data and preclinical data which are expected to be published soon this year, after which details of a clinical study will be announced.

Other active parts of its gene therapy pipeline include advancing research, manufacturing and regulatory activities for Fabry disease, with the goal of achieving Investigational New Drug (IND) designation by 2023. and to advance all studies and development activities for its Pompe disease program. Its AT-GAA therapy for Pompe disease has a target action date of July 29, 2022 for its biologics license application (BLA) and May 29, 2022 for the new drug application.

Amicus also noted that its oral precision drug Galafold (migalastat) is expected to maintain strong growth of between $350 million and $365 million in 2022 as it expands geographic coverage and continues Phase IV trials and registry initiatives. . Galafold is an oral treatment for Fabry disease in adults with favorable galactosidase alpha (GLA) gene variants. The drug is approved in more than 40 countries, including the United States, United Kingdom, Japan, and the European Union.

“We envision transforming Amicus into a leading global rare disease biotechnology company, led by two innovative therapies that we believe have a significant impact on the lives of people with Fabry and Pompe disease. This year, we will focus on continuing Galafold’s contribution to patients. worldwide and deliver the planned approval and launch of AT-GAA,” said Bradley Campbell, Amicus President and Chief Operating Officer, in a press release.

All of these details and more were revealed in its preliminary 2021 earnings and 2022 outlook report. to its Galafold global expansion and the global launch of AT-GAA to treat Pompe disease. Its pipeline of next-generation genetic drugs will also be carried out through its newly created research and development company, Caritas Therapeutics.