FDA Action Alert: Amicus, Vertex and TherapeuticsMD


This week had three important target action dates, known as Prescription Drug User Fee Act (PDUFA) dates, for the U.S. Food and Drug Administration (FDA). The agency got ahead of its schedule and approved all of these drugs. Let’s take a look.

Amicus Therapeutics and Migalastat for Fabry Disease

The FDA had a PDUFA date of Monday, August 13 for Amicus Therapeutics New Drug Application (NDA) for migalastat, called Galafold, for patients 16 years and older with Fabry disease who have amenable mutations. The drug has both Orphan Drug Designation and Fast Track Designation from the FDA, and Priority Review status. The FDA approved the drug on Friday, August 10.

Fabry disease is a rare, progressive, inherited lysosomal storage disorder caused by a deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), the result of mutations in the GLA gene. It causes specific lipids, mostly GL-3, to accumulate in the heart, kidneys, central nervous system, and skin. This can lead to pain, kidney failure, heart disease, and stroke. The disease affects about 3,000 people in the U.S.

Migalastat is a first-in-class chaperone therapy that has already been approved in the European Union as a monotherapy for Fabry disease in patients with the appropriate mutations. It has also been approved in Australia, Canada, Israel, Japan, South Korea and Switzerland. The drug stabilizes the body’s own dysfunctional enzyme so it can clear the accumulation of the lipids. The company’s lab test, the Galafold Amenability Assay, has classified more than 1,000 known GLA mutations as “amenable” or “not amenable” to treat with migalastat.

Reuters notes, “Amicus on Friday did not provide a U.S. price for Galafold. In an interview prior to the FDA’s approval, Chief Executive John Crowley said the company was committed to keeping new drugs priced at parity with or slightly below existing treatments, rather than charging a premium.”

Vertex Pharmaceuticals and Ivacaftor for Children Ages 12 to <24 Months for CF

Vertex Pharmaceuticals was awaiting the results of a supplementary New Drug Application (sNDA) for its cystic fibrosis (CF) drug ivacaftor in children ages 12 to <24 months. The PDUFA date was Wednesday, August 15. On August 7, the FDA approved the drug combination for use in children ages 2 through 5 years with CF who have two copies of the F508del-CFTR mutation. A Marketing Authorization Application (MAA) line extension for the same application has been submitted to the European Medicines Agency (EMA) with approval expected in the first half of 2019.

Ivacaftor, under the trade name Kalydeco, is approved for the treatment of CF in patients age 2 years and older who have at least one mutation in their CF gene that is responsible to the drug. Ivacaftor is also part of a combination therapy called Symdeko (tezacaftor/ivacaftor and ivacaftor) for the treatment of CF in patients aged 12 years and older who have two copies of the F508del mutation, or who have at least one mutation in the CF gene that is responsive to Symdeko.

TherapeuticsMD and its One-Year Vaginal Contraceptive System

The FDA had a PDUFA date of Friday, August 17 on whether to approve Boca Raton, Florida-based TherapeuticsMD’s segesterone acetate/ethinyl estradiol, one-year vaginal system for contraception, Annovera. But the agency approved it on Friday, August 10. The product is in the shape of a ring and combines a novel progestin, segesterone acetate (Nestorone) with a widely used estrogen to prevent ovulation for a full year, or 13 cycles. If approved, it would be the first and only procedure-free, reversible prescription contraceptive to offer a full year of protection against unintended pregnancy.

TherapeuticsMD entered into an exclusive license deal with the Population Council to commercialize the product in the U.S. on July 31, 2018. TherapeuticsMD will pay Population Council milestone payments of $20 million upon FDA approval and $20 million when the first commercial batch is released. Population Council is also eligible for milestone payments and royalties from commercial sales of the system. The Population Council was established in 1952 and is headquartered in New York City. It is a nongovernmental, nonprofit organization focused on stopping the spread of HIV and improving reproductive health through biomedicine, social science, and public health research in 50 countries.

“The U.S. contraceptive market is shifting toward long-acting solutions and we believe Annovera represents an exciting new entrant for women and healthcare providers by providing the first woman-controlled, procedure-free, long-acting, reversible birth control product putting the woman in control of both her fertility and menstruation,” said Brian Bernick, co-founder of TherapeuticsMD, in a statement. “We believe Annovera can help meet the needs of women who are looking for long-acting solutions, including women who have never given birth and women who are not in a monogamous relationship, who are often counseled not to use many of the currently available long-acting contraceptive products.”

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