Amicus Therapeutics FOLD announced that the FDA has granted accelerated approval to Galafold (migalastat) 123 mg capsules for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.


Shares are trading up on the approval news in pre-market trading. Year to date, Amicus’ shares have increased 6.6%, against the industry’s fall of 4.9%.

The drug was approved under the Subpart H Accelerated Approval pathway based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate.

Since the drug was approved under the accelerated approval pathway, Amicus will continue to study Galafold in a confirmatory phase IV program.

The FDA has approved Galafold for 348 amenable GLA variants. Per the company, Galafold is the first oral medicine for Fabry disease, and the first new therapy approved to treat Fabry disease in the United States in more than 15 years.

The approval was based on data from a phase III pivotal Fabry disease study in treatment-naïve patients, which demonstrated the efficacy of Galafold including a reduction in the amount of damaging disease substrate accumulated in the kidney capillaries.

Per estimates, there are approximately 3000 patients living with Fabry disease in the United States. Among these, 50% are currently untreated. Hence, the successful commercialization of the drug will boost the growth prospects of the company.

Galafold is already approved in European Union and Japan.

The news comes as a breather for investors as the road to approval in the United States has been bumpy.

The FDA earlier asked for additional data to approve the company’s application. However, last year, the Agency informed Amicus that it can submit an NDA. An additional phase III study as previously requested by the FDA to assess gastrointestinal (GI) symptoms was no longer required prior to an NDA submission.

However, the drug will face competition from Sanofi’s SNY Fabrazyme and Shire’s SHPG Replagal for the treatment of Fabry disease.

Zacks Rank & Stock to Consider

Amicus currently carries a Zacks Rank #3 (Hold).

A better-ranked stock in the healthcare sector is Gilead Sciences, Inc. GILD, sporting a Zacks Rank #1 (Strong Buy). You can see  the complete list of today’s Zacks #1 Rank stocks here.

Gilead’s earnings per share estimates have increased from $6.10 to $6.57 for 2018 over the last 60 days. Estimates for 2019 are also up by 14 cents.

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