Bangalore, 7 March 2025—The Karnataka High Court will hear arguments from cystic fibrosis (CF) patients and their families, who have petitioned the court to ensure access to a life-saving treatment. The treatment in question - a combination therapy of three CFTR modulators— elexacaftor, tezacaftor and ivacaftor are patented in India by the US pharmaceutical corporation Vertex Pharmaceuticals. This combination, marketed as Trikafta in high-income countries, is currently unavailable in India and is essential for the survival of CF patients.
Cystic fibrosis causes thick mucus build-up in the lungs, which can severely obstruct airways, lead to significant breathing difficulties, and shorten the life span of children with it. Thus, access to effective treatments is crucial for saving lives, and now patients can expect to live into adulthood and have normal lifespans with access to Trikafta.
Without effective treatment, patients with cystic fibrosis, many of whom are children, face the grim reality of premature death, with many patients dying before reaching their teenage years. Life-saving drugs like Trikafta, when administered early, could significantly extend their lives, offering them a near-normal life expectancy. The lack of access to such treatments raises serious concerns related to the right to life, as guaranteed under Article 21 of the Indian Constitution.
The petition highlights that despite Vertex holding multiple patents on Trikafta, the cystic fibrosis drug remains unavailable in the country. This has led to an abuse of the monopoly on the drug, preventing access for patients who desperately need it. The petitioners are calling for remedies that will address the non-working of the patent with generic competition, which would open up domestic production and supply, lower prices, and make the drug affordable for patients in India.
The parents, on behalf of their children, are asking the court to direct the government to license the drug for generic supply and facilitate domestic production of the medicine. They argue that experts have published data that generic versions of the drug could be manufactured for less than ₹6 lakh ($6000) per patient per year—98% less than Vertex’s price of $326,000 in high-income countries.
Parents of children of cystic fibrosis who have approached the Karnataka High Court for justice:
Anandi (name changed), the mother of a three-year-old daughter with CF (see photo), is one of the petitioners urging the courts to intervene and save her daughter’s life. "Without access to Trikafta, her life is a daily struggle. She undergoes constant medical treatments, including nebulisations, chest physiotherapy, and pancreatic enzyme replacement therapy. Trikafta could change this and save her life. Yet, the cost is staggering and unaffordable —more than ₹2.5 Crores annually if I import it from another high-income country where it is available. Pharmaceutical corporations are putting profits over people. We cannot afford to wait another decade for affordable treatment, watching our child’s strength slip away. We need our government, the courts, and lawmakers to recognise the human cost of not making affordable generics available and take action. Our health and our lives shouldn’t be held hostage by patent monopolies.”
Manisha (name changed), one of the parents of a teenager with CF who has approached the court, is concerned about delays in the court hearings. “We were importing the generic from Argentina, but the prices there have shot up to Rs 20 lakh for 3 months of drug supply due to political and financial crisis, and we cannot afford it anymore. Now, with no affordable options, we must seek a solution from the government to produce generic CF drugs locally. It's a matter of life and death, and it's time India steps up to ensure CF patients get the life-saving treatment they deserve", she adds.
“Vertex’s behaviour demonstrates a double standard in access to life-saving medications for rare diseases—selling the drug in high-income countries at exorbitant prices while ignoring the needs of those in low and middle-income countries, especially children with CF”, said Gayle Pledger, the parent of a child with cystic fibrosis (CF) and founder of the Right to Breathe Campaign that is advocating for the fundamental right to be able to breathe adequately, and this involves pushing for access to life-saving drugs by people living with cystic fibrosis.
Contact for interviews with CF patient families and experts:
Gayle Pledger - email: gayle@justtreatment.org tel: +44 790 521 7676
K M Gopakumar - kmgkumar@gmail.com tel: +919372927647
Notes to the Editor:
What is cystic Fibrosis and the Right to Breathe Campaign?
CF is a debilitating genetic disorder that significantly impacts various aspects of the body, primarily affecting the lungs and digestive system.
Cystic fibrosis causes thick mucus buildup in the lungs, which can severely obstruct airways and lead to significant breathing difficulties. Thus, access to effective treatments is crucial for saving lives.
Without effective treatment and care, patients with Cystic Fibrosis (CF) are dying—many of them young, often toddlers or children. Life-saving drugs known as CFTR modulators could significantly extend their lives if treatment is started early, offering near-normal life expectancy.
The most effective CF treatment combines three CFTR modulators (elexacaftor/tezacaftor/ivacaftor) that help the CFTR protein function correctly. This combination, co-packaged as tablets or granules and sold as Trikafta by Vertex in high-income countries, is life-saving for CF patients. This treatment delivers dramatic improvements in lung function, increases resilience to opportunistic infections, and reduces the need for hospitalisations while helping to avoid lung transplants and other invasive health interventions.
However, pharmaceutical corporations practice a double standard. While they sell rare diseases at high prices in high-income countries, they rarely register these life-saving medicines in low—and middle-income countries. At the same time, they apply for and get patent monopolies to block local production and supply from key generic manufacturing countries like India, which can meet the needs of poor patients in India and other low—and middle-income countries. Since the USFDA approved Trikafta in October 2019, Vertex’s revenue has touched $10 billion. Yet according to research published in the Journal of Cystic Fibrosis in 2022, only 12 per cent of CF patients worldwide have been prescribed the treatment,. whilst additional countries have subsequently gained access, the majority of countries are still denied access.
Generic competition from India has already demonstrated its ability to reduce prices. For example, in 2000, with generic competition, the cost of AIDS treatment fell from $10,000 per patient per year to $350 per patient per year, saving millions of lives in the developing world.
What is the relationship between patent monopoly and unavailability?
Once granted, patents are monopolies that allow pharmaceutical corporations to block competition from domestic manufacturers. India started granting such monopolies only in 2005 because they were mandated by the World Trade Organisation’s trade rules.
However, health safeguards in the Patents Act require the patent holder to make the drug available in the country.
The first product patent on the compound ivacaftor was granted on 20 April 2017 (IN282654), which expires in June 2025, but many additional evergreening patents were granted that extend the monopoly to 2028 and beyond. Tezacaftor is also patented in India, with the first patent, IN272379, granted on 30 March 2016, which expires in 2027. The elexacaftor product patent was granted on 17 January 2025 and expires only in December 2037. In addition, several evergreening patents extend the monopoly on the drug/s. In the Financial Times, Daniel Lyons, a portfolio manager at Janus Henderson, a top-25 Vertex shareholder, says the biotech faces “no real competitive threats” to its dominance in CF, with patents extending until at least 2039.
Despite holding patents for CF drugs in India, Vertex has not tried to seek marketing approval to make Trikafta available at an affordable price in India. Consequently, elexacaftor, tezacaftor, and ivacaftor are not covered for patients registered under the National Policy for Rare Diseases, which provides up to ₹50 lakh per patient for treatment at designated Centres of Excellence. Vertex’s failure to work the patent in India effectively denies CF patients access to this financial support to procure the drug.
In 2005, India designed its patent law with a key public health safeguard: it allows for issuing a compulsory license for generic manufacturing and supply if a patent is not being worked on and the ‘needs of the public’ are not being met. This safeguard can make life-saving medications affordable for all who need them in India and other resource-constrained settings. In this case, the first patent on one of the drugs in Trikafta was patented in 2017, and considering it was approved by the USFDA as way back as October 2019, more than six years have elapsed since the patents on Trikafta have not been worked.
What has happened in court to date?
The writ petition was filed in March last year before the Karnataka High Court. After much delay in the previous hearing on 30 January for cystic fibrosis patients (Ms X, Mr Y, and Ms Z* vs Union of India, WP 9920 of 2024), the Hon'ble High Court granted the Central Government two weeks to file their response.
*Names are anonymised to protect the identity of the children.