Amicus Therapeutics has announced that it has been given the
go-ahead to submit its new drug application (NDA), to the U.S FDA. The new drug,
migalastat, is an oral precision medication for Fabry disease. The drug is set
to be launched in the fourth quarter of 2017. The biopharmaceutical company is
gearing to submit the NDA under Subpart H, which grants them a faster approval.
Amicus Therapeutics will base the NDA on data it has collected from clinical
trials and previously available information, which shows a reduction in GL-3,
the disease-causing substrate. The data shows that the accumulation of GL-3 over
time causes the morbidity of Fabry disease, which is congruent with symptoms
like kidney failure, heart disease,
stroke and pain.
Amicus Therapeutics (NASDAQ: FOLD) is a biopharmaceutical
company with a global reach (YahooFinance). The company focuses on developing precision
medicine for rare and devastating disease brought about by genetic mutations. Amicus
Therapeutics has focused on rare diseases particularly of the kind collectively
known as lysosomal storage disorders. The company uses the Chaperone-Advanced
Replacement Therapy (CHART) to direct its product development. Many of the
products developed by the biopharmaceutical company are mostly enzyme
replacement therapies (ERTs). The company has been making ground-breaking
progress in its field of specialty and was recognized in 2014 as having the
largest collection of small molecule pharmacological chaperones in the
The company, based in Crabury New Jersey, has a second research facility in San
Diego. It has however faced some hard times along the way. One major setback
occurred in late 2009 when it ended a partnership with The Shire that jeopardized
their funding. However, the pharmaceutical company bounced back from the setback
and went on to make major headways in the development of curative techniques and
therapies for rare diseases thanks to new funding partners such as The Michael
J. Fox Foundation and the Alzheimer’s
Drug Discovery Foundation.
In 2013, the company, which had carved a reputation for itself as a pharmaceutical
entity that pushed boundaries, acquired Callidus Biopharma, thus getting
control of the ERT therapy treatment for Pompe disease.
Inthe same year, Amicus Therapeutics teamed up with GlaxoSmithKline and JCR Pharmaceuticals,
a partnership that lasted for three years in the period between 2010 and 2013.
The collaboration would handle research, investigating co-formulation with
recombinant alpha-galactosidase (JR-051).
Fabry disease affects a large spectrum of the population
Rare orphan diseases
caused by mutations may occur much more often than you may think. Numbers on
the ground show that more research and input towards the discovery of cures for
these conditions needs a major boost. According to statistics, Fabry effects 3,000
people in the U.S population. This presents Amicus Therapeutics with an
opportunity to effect significant change with the launch of its new drug, which
goes by the marketing name, Galafold.
Positive steps moving forward
John F. Crowley, Chairman, and CEO of Amicus Therapeutics expressed his
delight in the current state of affairs. In a statement to the media, he said
that the FDA had provided the right guidance for them to move forward in a
project that will change the lives of people living with Fabry disease in the
U.S. He said that it marked a sentimental milestone that could only fully be
enjoyed by the physicians, patients, and employees who have played a role in the
development of the new precision medicine.
According to Jay Barth, M.D., who is the Chief Medical Officer at Amicus
Therapeutics, the two main trials that were pivotal for the drug have been
successful and have earned its approval in the EU. The drug is also due to be
submitted for approval in other countries such as Canada, Japan, and Australia.
To Doctor Barth, the fact that the FDA is willing to review the drug shows that
indeed the company has achieved the gold standard in science-based therapy that
is patient-based. The doctor believes in the data the team has collected on all
aspects of the drug and is counting on it to bring relief to patients based in
commission (EC) has granted Amicus Therapeutics full approval to produce their
precision drug under the marketing name Galafold, as a therapy for long-term
treatment of Fabry disease for adults and adolescents from the age of 16. The
company also has also received positive marketing applications from countries
outside the EU such as Israel and Switzerland.
A little about Galafold
The therapy product is a first-of-its-kind chaperone that has had great
success in trials in countries across the European Union. The drug works by
stabilizing the dysfunctional enzymes from the body which then allows it to
clear the accumulation of disease substrate in patients with amenable
In a proprietary in vitro essay, a total of 800 known GLA mutations were
classified as either amenable or not amenable to medication with Galafold. The
EU label for such mutations includes 331 GLA mutations that have been
identified to be treated with Galafold. This represents 35% to 50% of the total
population currently dealing with Fabry disease.
The website, has been created for EU
practitioners to view information about Galafold to find out the mutations that
have been classified as amenable or not. Amicus Therapeutics is preparing to
submit some more updates concerning GLA mutations as more are tested by the
Galafold Amenability Assay.
The company has shown great character and strength in coming up with a
product that actually affects a part of the population. As most companies of the
kind have been known to do, drugs are usually watered down after the first few
batches are sold and sometimes fraud also creeps up. Amicus Therapeutics has
kept everything 100% ethical.
In recent years, the quantity of false medications has made entered the U.S market has
risen and Amicus Therapeutics has been encouraged all round to continue setting
new levels of achievement. The new drug, which is going to be a first in line
treatment, can only be given time to seep into the U.S medical system and
become a mainstay. Even though it is hard to speculate how much success this
one may garner, it is likely to become a go-to for many patients who have been
struggling with Fabry. It might also be some time before consumers decide to warm up to this wonder drug, but hopes are high in the industry, especially when you consider the fact that no other companies have made the sort of breakthroughs Amicus Therapeutics is currently making. It is also believed that the prices will be quite reasonable once the stakeholders involved find a way of lowering the costs of production.
More about Amicus Therapeutics on https://www.crunchbase.com/organization/amicus-therapeutics