CRANBURY, N.J., March 22, 2016 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a biotechnology company at the forefront of therapies for rare and orphan diseases, has initiated a reimbursed expanded access program (EAP) to provide Fabry patients who have amenable mutations with access to migalastat. The EAP will be implemented in certain territories where reimbursement can be secured prior to marketing authorization and commercial availability, beginning in France.
The French National Agency for Medicines and Health Products Safety (ANSM) has granted an "Autorisation Temporaire d'Utilisation dite de cohorte" (cohort ATU), a Temporary Authorization for Use for patient sales of migalastat for Fabry patients who have amenable genetic mutations who meet the criteria for the cohort ATU. An ATU is the regulatory mechanism used by the ANSM to make non-approved drugs available to patients in France when a genuine public health need exists.
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, “Consistent with our Company mission, we are committed to providing access to migalastat for Fabry patients with amenable mutations in countries where there is an available mechanism to secure reimbursement prior to approval. We are grateful to the French authorities for granting an ATU to allow Fabry patients in France to begin treatment with migalastat and we are pursuing additional opportunities for early access in other countries.”
The ATU for migalastat was granted after an assessment by the ANSM of a full application dossier that included clinical and safety data from clinical studies of migalastat. In France, patients with Fabry disease who have amenable mutations may receive treatment with migalastat before marketing authorization for the product is granted in the European Union. Government allocations to hospitals will allow payment for migalastat for patients included in the ATU program.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq:FOLD) is a biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, SD-101 for Epidermolysis Bullosa (EB), as well as novel enzyme replacement therapy (ERT) products for Fabry disease, Pompe disease, and other lysosomal storage disorders.
Forward-Looking Statements
This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of Amicus' candidate drug products, the timing and reporting of results from preclinical studies and clinical trials evaluating Amicus' candidate drug products and the projected cash position for the Company. Words such as, but not limited to, "look forward to," "believe," "expect," "anticipate," "estimate," "intend," "potential," "plan," "targets," "likely," "may," "will," "would," "should" and "could," and similar expressions or words identify forward-looking statements. Such forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. The inclusion of forward-looking statements should not be regarded as a representation by Amicus that any of its plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong. They can be affected by inaccurate assumptions Amicus might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress, timing and outcomes of discussions with regulatory authorities and the potential goals, progress, timing and results of preclinical studies and clinical trials, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in the business of Amicus, including, without limitation: the potential that results of clinical or pre-clinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities may not grant or may delay approval for our product candidates; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; the potential that we will need additional funding to complete all of our studies and, our dependence on third parties in the conduct of our clinical studies. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. With respect to statements regarding projections of the Company's cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2015. You 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 Amicus undertakes no obligation to revise or update this news release to reflect events or circumstances after the date hereof. This caution is made under the safe harbor provisions of Section 21E of the Private Securities Litigation Reform Act of 1995.
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