Symdeko Approved for Treatment of Cystic Fibrosis
February 13, 2018 – The U.S. FDA has approved Symdeko™ (tezacaftor/ivacaftor and ivacaftor), manufactured by Vertex, to treat certain patients who have cystic fibrosis.
Cystic fibrosis (CF) currently affects an estimated 30,000 Americans, with an estimated 1,000 new diagnoses of CF made annually. The genetic disorder interferes with the body’s ability to transport water and salts, leading to respiratory difficulties and inability to properly digest food. Due to improved treatments, approximately half of CF patients now survive to adulthood. An estimated 90% of CF patients have an F508del mutation.
Symdeko is indicated to treat CF in patients who are at least 12 years old and are homozygous for the F508del mutation. It is also indicated to treat CF patients who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor and ivacaftor, based on in vitro data and/or clinical evidence. An FDA-approved test for CF mutations must be used to verify the patient’s genotype prior to treatment with Symdeko.
A combination treatment, Symdeko consists of tezacaftor 100mg/ivacaftor 150mg tablets packaged along with Kalydeco® (ivacaftor) 150mg. Recommended daily dosing is one tezacaftor/ivacaftor tablet taken in the morning, and one Kalydeco tablet taken 12 hours later. Patients who received Symdeko in clinical studies “experienced statistically significant and clinically meaningful improvements in lung function and other measures of disease, with a favorable safety profile,” according to a press release from the manufacturer.
The estimated annual cost for Symdeko is $292,000. The product has already been launched.
Adverse reactions or quality problems experienced with the use of a recalled product may be reported to the FDA’s MedWatch Adverse Event Reporting Program either online, by U.S. mail or by fax.
- Complete and submit the report Online: www.fda.gov/medwatch/report.htm
- U.S. Mail or Fax: Download form www.fda.gov/MedWatch/getforms.htm or call 1-800-332-1088 to request a reporting form, then complete and return to the address on the pre-addressed form, or submit by fax to 1-800-FDA-0178
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