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K is for KALYDECO®
KALYDECO® (from Vertex Pharmaceutical Limited) uses ivacaftor for treating cystic fibrosis in people with certain mutations in the gene for a protein called cystic fibrosis transmembrane conductance regulator (CFTR).
ivacaftor
How it works:
Cystic fibrosis is caused by mutations in the CFTR gene. The CFTR gene produces a protein that regulates mucus and digestive juice production. These mutations lead to thick mucus and digestive fluids, causing blockages, inflammation, increased lung infection risk, and poor digestion and growth.
KALYDECO® contains the active substance ivacaftor. Ivacaftor enhances the activity of the defective CFTR protein, making mucus and digestive juices less thick and alleviating disease symptoms.
Marketing approval:
KALYDECO® is approved for marketing in the United States (approved by the FDA in January 2012 (tablet) and March 2015 (granule)) and Europe (approved by the EMA in July 2022).
Patent protection:
There are a number of patent rights protecting various aspects of KALYDECO® in the United States and Europe (as well as in many other countries around the world). For example:
European patent no. 1773816 relates to N-(5-hydroxy-2,4-ditert-butyl-phenyl)-4-oxo-1H-quinoline-3-carboxamide or a pharmaceutically acceptable salt thereof.
European patent no. 2489659 relates to a pharmaceutical composition comprising the compound N-(5-hydroxy-2,4-ditert-butyl-phenyl)-4-oxo-1H-quinoline-3-carboxamide, or a pharmaceutically acceptable salt thereof, wherein said composition further comprises: a) a pharmaceutically acceptable carrier or adjuvant; and b) an additional agent selected from a mucolytic agent, bronchodilator, an anti-biotic, an anti-infective agent, an anti-inflammatory agent, cystic fibrosis transmembrane receptor (CFTR) modulator, or a nutritional agent.
European patent no. 2464337 relates to a pharmaceutical composition comprising about 34.1 wt% of a solid dispersion by weight of the composition, wherein the dispersion comprises 80 wt% of substantially amorphous or amorphous N-[2,4-Bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide (Compound 1)
by weight of the dispersion, 19.5 wt% of hydroxypropylmethylcellulose succinate (HPMCAS) by weight of the dispersion, and 0.5 wt% sodium lauryl sulfate (SLS) by weight of the dispersion; about 30.5 wt% of microcrystalline cellulose by weight of the composition; about 30.4 wt% of lactose by weight of the composition; about 3 wt% of sodium croscarmellose by weight of the composition; about 0.5 wt% of SLS by weight of the composition; about 0.5 wt% of colloidal silicon dioxide by weight of the composition; about 1 wt% of magnesium stearate by weight of the composition; wherein the pharmaceutical composition is made into a tablet; for use in treating or lessening the severity of cystic fibrosis in a patient; wherein said use comprises administering the pharmaceutical composition concurrently with, prior to, or subsequent to one or more other desired therapeutics; wherein the patient possesses a cystic fibrosis transmembrane receptor (CFTR) with a ΔF508 mutation on both alleles; and wherein the other desired therapeutic is a CFTR modulator other than compound 1.
There are also US patents corresponding to each of the European patents described above.
Patent support from Secerna
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