Stuart A. Arbuckle
Executive Vice President and Chief Operating Officer at Vertex Pharmaceuticals
Thanks, Reshma. I'll first discuss CF and then as we're entering a new era of commercial diversification, provide some highlights of the ongoing CASGEVY launch and the outlook for suzetrigine in acute pain.
As Reshma noted, we once again delivered strong results in CF as we continue to grow the number of eligible patients receiving our CFTR modulators. First quarter year-over-year US growth was driven by continued strong performance of TRIKAFTA, including in patients ages two to five years old, following the approval in this patient population in April of last year. Outside the US, we also saw growth this quarter driven by the rollout of KAFTRIO in the EU in patients ages two to five, following approval in this age group in November 2023, and we will continue to drive access and uptake in more EU countries over the course of the year.
Our outlook in CF is bright in the short, medium and long-term. We will drive growth in the near-term by reaching more eligible patients, including younger age groups and additional geographies. For example, we recently received EU approval of KALYDECO in patients between the ages of one month, up to four months old. We also expect regulatory approvals for additional rare genotypes for KAFTRIO in the EU and TRIKAFTA in the US and Canada later this year.
And Brazil is a good example of a new geography. Up to now, some patients from Brazil have been able to benefit from our CFTR modulators through name patient sales. We recently secured government reimbursement for TRIKAFTA in ages six plus and are in the process of launching TRIKAFTA for all eligible patients there. We will then look to drive further CF growth over the medium-term with the vanzacaftor triple combination launch, as many existing TRIKAFTA patients may seek to achieve even greater levels of CFTR function with the added convenience of once-daily dosing. And there are also more than 6,000 patients who have discontinued one of our current CFTR modulators who may be interested in a new treatment option.
Furthermore, there are 31 additional rare mutations not previously responsive to our other CFTR modulators that are responsive to the vanzacaftor triple. Our launch preparations are well underway, including pre-approval information exchange with payers, and we are both encouraged by our interactions to date and excited by the opportunity to launch our fifth medicine in CF. Longer term, we expect continued growth in CF from our mRNA program, VX-522 for the more than 5,000 people with CF, who do not respond to CFTR modulators.
Now turning to CASGEVY, and our launches in sickle cell disease and beta thalassemia. We are making strong progress with ATC activation, physician and patient engagement and payer conversations. Enthusiasm from stakeholders is high in all regions, and our teams are working to translate this historic scientific achievement into meaningful patient benefit in the real world. Let me provide some insights on the launch with two key metrics we are sharing externally as important markers of our early launch progress. The number of activated authorized treatment centers or ATCs, and patient cell collections. Recall that Vertex will recognize revenue for CASGEVY near the end of the patient journey at infusion.
Starting with ATC activation. You may recall, we are prioritizing approximately 75 ATCs globally and already had nine ATCs activated at launch, even ahead of knowing the final label or pricing for CASGEVY. We are pleased with our progress as we now have more than 25 activated centers including centers in all regions where CASGEVY is approved. Even more important than the number of ATCs activated is patient initiations and cell collections. Many patients have begun the treatment journey, and as of mid-April, five patients already had cells collected. This is excellent progress given the short time frame since approval and the complexity and length of the patient journey. These cells collections have occurred across all regions where CASGEVY is approved, the US, Europe and the Middle East.
We also continue to make great progress with payers, who recognize the transformative clinical benefits of CASGEVY and are moving quickly to provide rapid and equitable access. In the US commercial market, we have contracts and/or published policies in place for over 200 million lives or nearly 65% of total lives. In the government Medicaid sector, we have policies in place or active contract negotiations ongoing with 18 states. And in the meantime, all states have confirmed their intent to provide case-by-case coverage.
Outside the US, we are also making progress with reimbursement and access, either through formal reimbursement agreements or early access programs. In Europe, we see strong traction in France with a reimbursed early access program in TDT. We're particularly pleased with our progress in the Middle East, which is a new region for Vertex and especially important for CASGEVY given the high prevalence of sickle cell disease and the government's clear focus on elevating the health of their citizens.
Since receiving regulatory approvals from KSA and Bahrain, we have worked with local health care authorities and refined our epidemiology estimates for the region. Our work indicates that the eligible 12-plus sickle cell disease and beta thalassemia population in KSA and Bahrain that we could serve is in excess of 23,000 patients, a potentially larger opportunity than even the US. These regions have the infrastructure to administer medicines like CASGEVY given the prevalence of the diseases and relatively high volume of allogeneic stem cell transplants performed annually.
And importantly, we have already secured reimbursement agreements in KSA and Bahrain, allowing certain eligible patients to access CASGEVY for both sickle cell disease and transfusion dependent thalassemia. In addition to having activated ATCs and collected cells from our first patients in the Middle East, we continue to work with local health care professionals to increase the number of ATCs and expand patient access in the region.
Shifting now to suzetrigine. We believe this highly selective Nav1.8 pain signal inhibitor has the potential to provide a transformative treatment option for the millions of patients suffering from acute and peripheral neuropathic pain. This quarter, I'm going to limit my commercial comments to the opportunity in acute pain.
Throughout its clinical trials to date, suzetrigine has shown a compelling combination of efficacy and safety with strong potential to be used across a range of moderate to severe acute pain conditions, both surgical and non-surgical and across a range of settings. This profile will ideally address the clear unmet need among both patients and physicians, effective pain relief with a favorable safety and tolerability profile.
On prior investor webcast, we provided details on this opportunity, including the magnitude, approximately 80 million patients are prescribed a medicine for moderate-to-severe acute pain each year in the US and the high concentration with approximately two-thirds of patients being treated in the institutional setting. There is further concentration within that setting in approximately 2,000 institutions that roll up to around 150 IDNs. Accordingly, they can be served with a specialty commercial infrastructure.
We have also detailed the mix of settings for their over 1 billion calendar days of acute pain treatment, 15% are prescribed and dispensed in an institutional setting. 35% are prescribed discharge and 50% are prescribed in physicians' offices. This quarter, I'll provide you with some insights on our go-to-market strategy and an update on the legislative and payer landscape. We are focused on the institutional setting, given these approximately 2,000 institutions account for 50% of acute pain prescriptions.
Extensive market research has also helped us identify an initial set of specific acute pain conditions and procedure types with high clinical fit, such as high-volume surgical procedures, pain conditions that typically require prescription pay medicines at discharge or where we can seek to replace or significantly reduce opioid utilization. And the related physician specialties that are likely to adopt and champion suzetrigine.
The key health care professionals we will be targeting include orthopedic, general and plastic surgeons, emergency department physicians, anesthesiologists and pain medicine specialists. Given the dynamics for new medicines to be approved for use in institutions, we expect the earliest uptake of suzetrigine will occur at discharge. Recall, this discharge segment represents roughly 35% of the approximately 1.1 billion calendar days of acute pain treatment in the US each year. The average prescription length in this setting is approximately two weeks. Treatment in this setting commonly includes opioids where prescription length is shorter four to five days due to side effect profile, addiction concerns and prescribing limits at the state and IDN and hospital level.
We are already engaging with key decision-makers across the formulary and access landscape, including pharmacists, PBMs, payers, IDNs and GPOs. We expect these stakeholders to make formulary and coverage decisions throughout the first year of the launch and thus plan to engage in contracting discussions in the second half of this year, ahead of launch to support the potential for accelerated formulary adoption.
We've also made great progress in the build-out of our commercial team. Our field leadership team are now on board and fully trained and have engaged in the hiring of the field force until after the Phase 3 data we are now finalizing the hiring of 150 new customer-facing colleagues.
Finally, we know the significance of policy in the world of pain treatment with important legislation like the NOPAIN Act already on track for implementation in 2025 and bills like the alternatives to PAIN Act recently introduced. Our long-standing efforts continue to help shape state and federal policy initiatives to: one, encourage consideration and use of non-opioid alternatives; and two, remove financial barriers to choosing a branded non-opioid.
Overall, we plan for a high science, digitally enabled commercialization approach with a strong focus on population health decision makers. In addition, both patient advocacy and public policy efforts complement and supplement our commercial activities.
In conclusion, it's an exciting time to be at Vertex. We continue to treat more CF patients around the world and are well advanced in planning for the launch of the vanzacaftor triple combination. We are entering a new era of commercial diversification with the launch of CASGEVY in the US, Europe and the Middle East. And our launch preparations for suzetrigine in acute pain are well underway as we seek to fundamentally redefine the treatment of pain and drive further diversified revenue growth.
I'll now turn the call over to Charlie to review the financials.
