FDA expands approval for Vertex Pharmaceuticals’ cystic fibrosis drug
WASHINGTON — The Food and Drug Administration on Wednesday expanded the approved use of Vertex Pharmaceuticals’ Kalydeco (ivacaftor) for treating cystic fibrosis. According to the FDA, the approval triples the number of rare gene mutations that the drug can now treat, expanding the indication from the treatment of 10 mutations, to 33, adding it based its decision, in part, on the results of laboratory testing, which it used in conjunction with evidence from earlier human clinical trials. The approach provides a pathway for adding additional, rare mutations of the disease, based on laboratory data.
“Many rare cystic fibrosis mutations have such small patient populations that clinical trial studies are not feasible,” said Dr. Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research. “This challenge led us to using an alternative approach based on precision medicine, which made it possible to identify certain gene mutations that are likely to respond to Kalydeco.
Cystic fibrosis affects the cells that produce mucus, sweat and digestive juices. These secreted fluids are normally thin and slippery due to the movement of sufficient ions (chloride) and water in and out of the cells. People with the progressive disease have a defective cystic fibrosis transmembrane conductance regulator gene that can’t regulate the movement of ions and water, causing the secretions to become sticky and thick. The secretions build up in the lungs, digestive tract and other parts of the body leading to severe respiratory and digestive problems, as well as other complications such as infections and diabetes.
Results from an in vitro cell-based model system have been shown to reasonably predict clinical response to Kalydeco. When additional mutations responded to Kalydeco in the laboratory test, researchers were thus able to extrapolate clinical benefit demonstrated in earlier clinical trials of other mutations. This resulted in the addition of gene mutations for which the drug is now indicated.
Kalydeco, available as tablets or oral granules taken two times a day with fat-containing food, helps the protein made by the CFTR gene function better and as a result, improves lung function and other aspects of cystic fibrosis, including weight gain. If the patient’s genotype is unknown, an FDA-cleared cystic fibrosis mutation test should be used to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use.
Cystic fibrosis is a rare disease that affects about 30,000 people in the United States. Kalydeco is indicated for patients aged 2 and older who have one mutation in the CFTR gene that is responsive to drug treatment based on clinical and/or in vitro (laboratory) data. The expanded indication will affect another 3 percent of the cystic fibrosis population, impacting approximately 900 patients. Kalydeco serves as an example of how successful patient-focused drug development can provide greater understanding about a disease.
Common side effects of Kalydeco include headache; upper respiratory tract infection (common cold) including sore throat, nasal or sinus congestion, or runny nose; stomach (abdominal) pain; diarrhea; rash; nausea; and dizziness. Kalydeco is associated with risks including elevated transaminases (various enzymes produced by the liver) and pediatric cataracts. Co-administration with strong CYP3A inducers (e.g., rifampin, St. John’s wort) substantially decreases exposure of Kalydeco, which may diminish effectiveness, and is therefore not recommended.
Consumer Reports research pinpoints reasons for rising drug prices
NEW YORK — High-deductible insurance plans, changes to “your formulary,” and a few “extremely high-priced drugs” are the main culprits behind increasing drug prices, according to Consumer Reports data released Tuesday.
“An estimated 28 million Americans — or 25% of those who regularly take prescription medications — saw a price increase in the past 12 months, according to the latest Consumer Reports “Best Buy Drugs” nationally representative survey of nearly 1,000 adults who regularly take prescription medication,” the magazine revealed.
The magazine acknowledged rising drug costs are not a new problem. It also provided strategies for consumers seeking to rein in drug costs. This is the advice the publication provided:
- Do a drug review with your doctor or pharmacist;
- Consider nondrug options for what ails you;
- Try a cheaper alternative;
- Don't always use your insurance;
- Ask for a lower price at the pharmacy; and
- Shop around.
To read the full report, click here.
J&J: 10 products with “blockbuster potential” to be filed by 2021
NEW BRUNSWICK, N.J. — Johnson & Johnson and its Janssen Pharmaceutical Cos. will announce plans to launch or file for regulatory approval more than 10 new products with “blockbuster potential” between 2017 and 2021, as well as 50-plus line extensions of new medicines, senior executives said Wednesday during an industry analysts meeting.
Among the late-stage blockbusters projected to file for regulatory approvals between 2017 and 2021, include:
- apalutamide (ARN-509) for pre-metastatic prostate cancer;
- esketamine for treatment-resistant depression;
- talacotuzumab (CSL362) for acute myeloid leukemia;
- erdafitinib (FGFR Inhibitor) for solid tumors;
- niraparib for prostate cancer;
- imetelstat for myelofibrosis;
- pimodivir (JNJ-3872) for influenza A;
- lumicitabine (JNJ-1575) for respiratory syncytial virus (RSV) infection; and,
- JNJ-7922 (orexin-2 antagonist) for adjunctive treatment for major depressive disorder.
The company also said it will share its plans to continue driving sustainable growth by leveraging its strong portfolio of core blockbuster products, the “industry-leading” productivity of its innovation model, and the pending acquisition of Swiss-based biotech company Actelion.
“With a growing core business of differentiated medicines and a strong line-up of innovative products expected to launch or file over the next five years, we are leading the industry in advancing the health of patients around the world,” said Alex Gorsky, chairman and CEO. “Our pharmaceutical business will continue to be a significant driver of innovation and growth for Johnson & Johnson. With our proven global commercial capabilities and robust pipeline, we are well-positioned to continue delivering strong, long-term, sustainable growth.”