A drug treatment for Cyctic Fibrosis
was approved by FDA. The drug was developed after a gene responsible
for causing Cycstic Fibrosis was found. The identification of gene has
been acomplished after almost 22 years.
Vertex is producer of the drug named as Kalydeco. Cystic fibrosis is caused because of defective gene responsible for decoding a protein known as Cystic Fibrosis Transmembrane Regulator. It is found that the gene is behind 4% cases of cystic fibrosis.
Approval of Kalydeco is believed to be of great significance as all other available treatments in market are only capable to treat symptoms of disease. A press note has been published by USA FDA regarding the approval.
The treatment is approved for patients of age above six and with confirmed G551D mutation. Vertex is quite please as approval came earlier than expected.
Considering rareness of the disease, producer company is contemplating to initiate programs to make the drug available at affordable cost or may be for free. Current estimation of drug cost is $294,000 per year which is near to the cost of other treatment for same disease.
Kalydeco is expected to generate millions of dollars in US market alone. There are 200,000 patients of Cystic Fibrosis in USA.
Recent amendment in FDA approval policy is the reason that the drug was approved with priority in review.
Clinical trial included 213 patients with G551D defect. The pill was administered twice a day in all participants. During trial Kalydeco showed some side effects such as stomachache, headache, infection in respiratory system and diarrhea.
Cystic Fibrosis is serious disease as it decreases life of patients because of thick mucus accumulated in digest tract and lungs. Patient suffering from CF lives upto 36-38 years.
Vertex is producer of the drug named as Kalydeco. Cystic fibrosis is caused because of defective gene responsible for decoding a protein known as Cystic Fibrosis Transmembrane Regulator. It is found that the gene is behind 4% cases of cystic fibrosis.
Approval of Kalydeco is believed to be of great significance as all other available treatments in market are only capable to treat symptoms of disease. A press note has been published by USA FDA regarding the approval.
The treatment is approved for patients of age above six and with confirmed G551D mutation. Vertex is quite please as approval came earlier than expected.
Considering rareness of the disease, producer company is contemplating to initiate programs to make the drug available at affordable cost or may be for free. Current estimation of drug cost is $294,000 per year which is near to the cost of other treatment for same disease.
Kalydeco is expected to generate millions of dollars in US market alone. There are 200,000 patients of Cystic Fibrosis in USA.
Recent amendment in FDA approval policy is the reason that the drug was approved with priority in review.
Clinical trial included 213 patients with G551D defect. The pill was administered twice a day in all participants. During trial Kalydeco showed some side effects such as stomachache, headache, infection in respiratory system and diarrhea.
Cystic Fibrosis is serious disease as it decreases life of patients because of thick mucus accumulated in digest tract and lungs. Patient suffering from CF lives upto 36-38 years.
No comments:
Post a Comment