Amicus Therapeutics Introduces Galafold for Fabry Disease Patients

Fabry disease can affect any male or female in any ethnic group who carries a faulty inherited gene, but Amicus Therapeutics has a drug that can treat it. The U.S. FDA is accepting the company’s fourth quarter of 2017 submission of a new drug application (NDA) for migalastat. The disease builds up a fatty substance that can damage the brain, heart, kidneys, nervous system and skin by narrowing blood vessels.


Understanding the Work of Amicus Therapeutics

As a global biotechnology company, Amicus Therapeutics maintains a position at the forefront of research that develops therapies for orphan and rare disease. With a focus on human genetic diseases, the company maintains a steady flow of research projects that produce advanced treatments, including one that may help people who suffer from Fabry disease. A benefit of the NDA is its potential for breaking down substances such as fatty acids and oils.


The disease occurs when the enzyme that can perform that function naturally does not work correctly in the body. Dermatologists in Germany discovered and named the condition at the end of the 19th century, and Amicus Therapeutics offers a way to confront it more than 100 years later. Approximately 3,000 Americans have a diagnosis of Fabry disease. As the single largest geographic area with a concentration of patients with the condition, it provides an opportunity for Amicus Therapeutics to exert an impact on those who have amenable mutations.


Seeking Accelerated Approval

The FDA offers three paths that can expedite approval of a new drug or biologic that has the potential to treat serious diseases, especially those that provide a new way to do so.


Amicus Therapeutics is using one of them to obtain accelerated approval for migalastat, and it plans to use the existing data that it developed in clinical studies, some of which relate to a reduction in the disease-causing substrate (GL-3). Researchers at the company believe that the progressive accumulation of the substrate may lead to the dire consequences of Fabry disease that include pain, heart disease, stroke and kidney failure.

Reviewing Supporting Statements from Officials

The Chairman and Chief Executive Officer of Amicus Therapeutics, John F. Crawley, expressed his approval of the guidance from the FDA that presents a huge advance for the thousands of Fabry patients in the United States. The company is “moving ahead expeditiously” to submit the NDA and “accelerate the U.S. pathway” for the product, he said. The efforts of physicians, patients and employees of Amicus Therapeutics deserve credit for their perseverance and dedication in developing the “precision medicine,” Crawley stated.


Citing the significance of data from the company’s clinical trials, Jay Barth, M.D., the Chief Medical Officer of Amicus Therapeutics, stated that it has already provided a basis for approvals for migalastat by authorities in Switzerland, Israel and the EU. The product has pending regulatory submissions in Australia, Canada and Japan, according to Barth. He described the data that supports the drug as the “gold standard.”


The Founder and Executive Director of the Fabry Support and Information Group, Jack Johnson, expressed satisfaction with the plan to submit an NDA as soon as the fourth quarter. His awareness of the need for treatment choices that do not currently exist makes him look forward to getting “one step closer to a new oral therapy,” he said. He praised Amicus Therapeutics for serving as a “true partner” for a community of patients who have amenable mutations.


Getting to Know Amicus Therapeutics

The company does a commendable job of leveraging its technology into a positive force against human genetic diseases. Mutated proteins get particular attention from the scientists at the global biotechnology company that tackles rare diseases. Its leading candidate for FDA approval, migalastat, offers a customized and personalized drug that can treat patients who have a diagnosis of Fabry disease.


Ample reasons justify the company’s pride in a drug that has potential as a first-to-market therapy for patients who have no alternative but to suffer from the effects of the disease. Amicus Therapeutics understands the difficulties that patients who have the rare genetic connective tissue disorder face, and it devotes sound scientific research to find a medicine that can help them. A statement from Amicus Therapeutics expresses its belief in fighting to “remain at the forefront of therapies for rare and orphan diseases.” The company seeks to deliver the best treatments for people who live with these conditions and to embrace constant innovation.


Defining the Terms

Patients who have amenable genetic mutations are potential candidates for using migalastat to stabilize a dysfunctional enzyme. Such mutations respond to therapy with the drug based on a proprietary in vitro assay. EU healthcare providers may access information about amenable or not amenable mutations at the Galafold website, the marketing product name for migalastat. The company estimates that up to 50 percent of patients globally fit that description. The European Commission approves the drug as the first line of treatment for patients who have a confirmed diagnosis of the disease and who have an amenable mutation. The company offers an audio webcast at the Investors section of the corporate website.


Examining the Recommendations

Patients who use Galafold need supervision by doctors who have experience in diagnosing and treating Fabry disease and can provide advice about dosages and side effects. Prescribing information describes contraindications to the use of Galafold, including hypersensitivity to its active substance or potential reactions to other ingredients. A headache is a common adverse reaction that occurs in about 10 percent of patients who use Galafold.


Situations to Avoid

Conditions that make usage inappropriate include renal impairment ( < 30 mL/min/1.73 m2), and it is not an appropriate choice for concomitant use with enzyme replacement therapy. Without a confirmation of an amenable mutation, patients need to avoid taking the drug. Testing has not established the safety and efficacy of the product for children aged 15 and younger.


Pregnant women need to check with a doctor, nurse or pharmacist about the use of the medication as very little experiential information exists. Female patients need to make sure to take effective birth control because science does not yet indicate the presence or absence of the drug in human milk.


Situations to Observe

Use of the drug requires periodic monitoring of real function, biochemical markers and echocardiographic parameters. In the case of an overdose, patients need to seek medical care.

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