Stuart A. Arbuckle
Executive Vice President and Chief Commercial and Operations Officer at Vertex Pharmaceuticals
Thank you, Reshma. I'll begin by reviewing the Q2 revenue performance of our CF medicines. Our Q2 global revenues reached nearly $1.8 billion, driven by increasing revenues outside the U.S. as a result of the launch of KAFTRIO and continued strong performance in the U.S. In the U.S., the launch of TRIKAFTA in ages 12 plus has been highly successful. And just under two years since regulatory approval, the vast majority of eligible patients have initiated treatment. We have continued to see very high persistence and compliance levels, and we're now focused on the ongoing launch of TRIKAFTA in children ages six to 11 in the U.S., following the approval in June.
Outside the U.S., we have made significant progress with reimbursement for our medicines. We now have reimbursed access for KAFTRIO in more than 15 countries outside the U.S., less than one year following EMA approval. This compares favorably to the industry standard, both in terms of the timeline in individual countries, and the total number of markets in which we have reimbursement agreements.
Our rapid reimbursement progress can be attributed to multiple factors, the transformative clinical benefits of the triple combination, the support and collaboration of governments and the CF community, and the expertise of our commercial team, built over the course of a decade. Importantly, as Reshma mentioned earlier, we are achieving reimbursement for our medicines at levels that recognize their considerable clinical value.
The pivotal clinical trial data for TRIKAFTA were unprecedented. And as with all of our prior medicines, we are continuing to track the long-term performance of the triple combination in extension studies of our pivotal trials and in the real world, to be able to fully understand and communicate the long-term benefits of CFTR modulators therapy to all key stakeholders.
We have previously shown powerful evidence that treatment with KALYDECO, ORKAMBI and SYMDEKO slows lung function decline, results in multi system benefits and transforms the course of the disease in CF patients. Earlier this month, we obtained our first long-term follow-up data with the triple combination. In the FF and FMF patient populations treated for at least 96-weeks with TRIKAFTA in the open label extension of the pivotal clinical trials, we do not see any decline in main lung function over time. This is a first for any of our CFTR modulators. We look forward to sharing these data in a forthcoming medical forum.
Although, we are pleased with what we've achieved so far, we still have a long way to go to reach all CF patients. As we have previously communicated, we estimate there are 83,000 people living with CF in the U.S., Canada, Europe and Australia. And approximately 90% of these are likely to benefit from a CFTR modulator. We estimate that there are more than 30,000 patients who could benefit from our current CF medicines, who are not yet being treated.
Reaching these patients which will drive significant additional revenue growth will be achieved by successfully launching our medicines where we have reimbursement, securing additional new reimbursement agreements and label expansions to younger age groups. And we remain confident we will be able to reach the vast majority of these patients.
I would now like to provide some perspective on the market opportunities for some of the other medicines in our mid and late stage pipeline. Starting with CTX001. With the amendment of our collaboration agreement with CRISPR Therapeutics, Vertex now has taken global leadership for all aspects of the CTX001 program. As a result, we are in a position to leverage Vertex's demonstrated ability to develop and secure access and reimbursement for transformative medicines.
We see tremendous potential for CTX001. We estimate that there are more than 150,000 patients in the U.S. and Europe, who have beta thalassemia, or sickle cell disease, approximately 32,000 of whom have severe disease. 25,000 are severe sickle cell disease patients, and the vast majority of these are in the U.S.
We believe that a gene editing approach which holds the potential for a one-time curative treatment will be highly valued by patients, physicians and payers. Consistent with our own internal market research, published physician surveys in the U.S. consistently indicate that they expect a quarter to a third of their sickle cell disease patients would be good candidates for a one-time curative approach using the current conditioning regimen, which is in line with the estimates of the numbers of severe patients.
And with gentler conditioning regimens in the future, we expect CTX001 to be an attractive option for a much larger proportion of the 150,000, beta thalassemia, and sickle cell disease patients. Our pre-commercial efforts are well underway. There are a number of notable features that are guiding our approach to this market, including one, patients with severe beta thalassemia and sickle cell disease are symptomatic and have a lifelong history of hospitalizations and other significant cost burdens. We are developing health economic models to demonstrate the cost effectiveness of a functional cure for these patients.
Two, new and flexible payment models will be needed for a functional cure for these diseases. We are engaged with payers to understand what models work best for them, and for patients. And three, patients are geographically concentrated. For instance, 75% of eligible sickle cell patients in the U.S. live in 15 states. We will use these and other insights to establish a commercial operation that is both lean and highly effective as we have in CF today.
Let me now turn to pain, and share our perspective on the market opportunity for a novel medicine in this area. Acute pain therapies represent 1.8 billion treatment days a year in the U.S. Despite more than 90% of prescriptions being generic, acute pain still represents a $4 billion market, underscoring the opportunity for a novel transformative agent. A new medicine that even takes a portion of the current treatment days has multibillion dollar potential. In acute pain, a significant component of the market is opioids. A medicine with high efficacy, and without the limitations of opioids, particularly their addictive potential would be transformative for patients and the healthcare system.
With regards to commercialization, treatment is highly concentrated within hospital and post-operative settings. 25% of U.S. hospitals account for 80% of all opioid prescriptions. Given this concentration, successful commercialization will be possible with a small specialist focused commercial model, again, consistent with our lean SG&A approach. Finally, type one diabetes, VX-880 has advanced a patient studies and relatively soon we will begin to have a view its clinical profile. We estimate that 60,000 patients in the U.S. and Europe with severe type one diabetes or type one diabetes with prior kidney transplant would be potential candidates for VX-880.
In and of itself, the islet cells alone program is a significant market opportunity. And existing transplant approaches establish a basis for the high value of a transformative therapy. Beyond VX-880 the cells and device program could address the broader type one diabetes population, 2.6 million patients in the U.S. and Europe.
In conclusion, it's an exciting time to be at Vertex as we continue to bring our CF medicines to more patients. And we still have significant growth ahead in CF, with new reimbursement agreements and label expansions to younger age groups. And beyond CF, our mid and late stage pipeline is rapidly advancing with each program having both the potential to transform lives and presenting a significant commercial opportunity.
And with that, I'll hand it over to Charlie.