Migalastat

What is Migalastat?

It is an FDA approved medication used in some patients with Fabry disease with a particular gene variant called galactosidase alpha.

What are the uses of Migalastat?

Galafold is an alpha-galactosidase a (alpha-gal a) pharmacological chaperone indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (gla) variant

How does Migalastat work?

Migalastat is a pharmacological chaperone that reversibly binds to the active site of the alpha-galactosidase A (alpha-Gal A) protein (encoded by the galactosidase alpha gene, GLA), which is deficient in Fabry disease. This binding stabilizes alpha-Gal A allowing its trafficking from the endoplasmic reticulum into the lysosome where it exerts its action.

Who Should Not Use Migalastat?

No contra-indications

Is Migalastat FDA approved?

Yes.

How should Migalastat be used?

• The recommended dosage regimen of galafold is 123 mg orally once every other day at the same time of day. Do not take galafold on 2 consecutive days. Swallow capsules whole; do not cut, crush, or chew the capsules.
• Take on an empty stomach. Do not consume food at least 2 hours before and 2 hours after taking galafold to give a minimum 4 hours fast.
• If a dose is missed entirely for the day, take the missed dose only if it is within 12 hours of the normal time that the dose should have been taken. If more than 12 hours have passed, resume taking galafold at the next planned dosing day and time and according to the every-other-day dosing schedule.

What are the dosage forms and brand names of Migalastat?

Capsules: 123 mg migalastat.

What side effects can Migalastat cause?

Most common adverse drug reactions ≥ 10% are:

• Headache
• Stuffy or runny nose and sore throat
• Urinary tract infection
• Nausea
• Fever

What special precautions should I follow?

None

What to do in case of emergency/overdose?

• In case of overdose, call the poison control helpline of your country. In the United States, call 1-800-222-1222.

• Overdose related information is also available online at poisonhelp.org/help.
• In the event that the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services. In the United States, call 911.

Can Migalastat be used in pregnancy?

The data is insufficient to assess the safety in pregnant women. However, there is a study that collects data on pregnant women with Fabry disease, either exposed or unexposed to GALAFOLD. Healthcare providers are encouraged to register patients or obtain additional information by contacting the Pregnancy Coordinating Center at 1-888-239-0758, email fabrypregnancy@ubc.com, or visit www.fabrypregnancyregistry.com.

Can Migalastat be used in children?

It is approved for use in adults although physicians can sometimes use it off label in children and adults

What are the active and inactive ingredients in Migalastat?

• Active ingredient: migalastat hydrochloride
• Inactive ingredients: magnesium stearate and pregelatinized starch. Capsule shells contain gelatin, indigotine - fd&c blue 2, and titanium dioxide. The black ink contains black iron oxide, potassium hydroxide, and shellac.

What should I know about storage and disposal of Migalastat?

• Store galafold at room temperature between 68°f to 77°f (20°c to 25°c).
• Keep galafold capsules in the blister card they come in to protect from moisture.
• Keep galafold and all medicines out of the reach of children.

Who manufactures and distributes Migalastat?

• Amicus therapeutics us, llc 3675 market street Philadelphia, pa 19104

• Dailymed label info on Migalastat
• FDA Migalastat

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