Cystic fibrosis patients live on borrowed air. Patients with this genetic disorder have thicker than normal mucus form in their lungs, pancreas and other organs that blocks their airway leaving them gasping for air.
Movies and tv shows that have depicted this disease into mainstream pop culture including Netflix's (NFLX) "Five Feet Apart," which released in 2019, can be hard to watch.
Decades ago most cystic fibrosis patients were lucky to live into their 20s. But medical breakthroughs have managed to change that dramatically.
Boston-based biotech firm Vertex Pharma's research and cystic-fibrosis gene therapies have been instrumental in helping patients with this exhausting disease live comfortably for decades longer.
Nearly 16,000 cystic fibrosis patients that have been treated with this drug -- Trikafta -- have seen an 87% reduction in the risk of lung transplant, a 77% reduction in pulmonary exacerbations, and a 74% reduction in the risk of death, the company said.
Blockbuster Drug
In November 2019, the Food and Drug Administration approved the company's fourth cystic fibrosis therapy, Trikafta, the drug has managed to cure patients without deteriorating their lung capacity, the company said.
Trikafta has steadily clocked revenues in excess of $1 billion, every quarter, starting from the fourth quarter of 2020. The three-drug cocktail, closed 2021 with a total revenue of $5.7 billion, accounting for 75% of Vertex Pharma's overall revenue.
Last year, Vertex Pharma expanded its leadership in cystic fibrosis-- treating more patients than ever before and advancing its next-in-class triple regimen into pivotal studies, the company said.
The drug was cleared to also be administered to children between 6-11 in the U.S.
Trikafta is now approved and reimbursed or accessible in more than 20 countries, according to the company's latest earnings statement.
"In October of 2019, Trikafta set a high bar in terms of both clinical trials and real-world data and has become the standard of care for patients with CF today," said Vertex Pharma President and Chief Executive Reshma Kewalramani in the company's latest earnings call transcript.
However, there are more than 25,000 patients remaining who are not yet treated including in countries like Canada, Spain and the Netherlands. Additionally, patients in geographies where Vertex Pharma has not yet been reimbursed such as Australia.
Trikafta's patents are protected until 2037, Vertex said in its annual financial report to the Securities and Exchange Commission (SEC). In other words, the biotech firm does not fear a generic competitor for at least 15 years.
Lots of Income Opportunities
The company also wants to help many more younger patients through its ongoing label expansions and continues to make progress in this segment.
"For the 90% of cystic fibrosis patients who can benefit from a CFTR modulator, we see continued significant growth ahead as we have more than 25,000 patients who could benefit from Trikafta and our other cystic fibrosis medicines but who are not yet on treatment.
"Nonetheless, if it is possible to deliver better clinical outcomes in Trikafta, we are determined to be the ones who do so," Kewalramani added.
In collaboration with Moderna (MRNA), Vertex is evaluating cystic fibrosis mRNA therapeutics designed to treat the underlying cause of cystic fibrosis by enabling cells in the lungs to produce functional CFTR [cystic fibrosis transmembrane conductance regulator] protein for the treatment of the close to 10% of patients who do not produce this protein.
IND-enabling studies are underway, and the company plans to submit an IND for this program in 2022. The purpose of IND-enabling studies is to secure approval to conduct the first-in-human clinical trials with a new drug.
Vertex Pharma has estimated total product revenues of $8.4 million to $8.6 billion for the full year 2022, 12% over last year.
