Vertex Announces that Inaxaplin (VX-147) has been granted Breakthrough Therapy Designation by the US FDA and Priority Drug Designation (PRIME) by the EMA

– Vertex has granted nine Breakthrough Therapy designations and three PRIME designations in its pipeline programs to date –

BOSTON–(BUSINESS WIRE)–Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the United States Food and Drug Administration (FDA) has granted inaxaplin (VX-147) treatment designation Breakthrough for APOL1-mediated Focal Segmental Glomerulosclerosis (FSGS) and The European Medicines Agency (EMA) has granted inaxapline Priority Medicine Designation (PRIME) for APOL1-mediated Chronic Kidney Disease (AMKD) . Inaxaplin is the first experimental therapy aimed at treating the underlying cause of AMKD.

FDA Breakthrough Therapy designation is intended to expedite the development and review of drugs intended to treat a serious condition with preliminary clinical evidence indicating that the drug may demonstrate substantial improvement over existing treatments on one or more clinically significant. Breakthrough Therapy designation was granted based on the Phase 2 clinical study of inaxapline in patients with APOL1-mediated FSGS, a form of AMKD.

EMA’s PRIME designation is a regulatory mechanism that provides early and proactive support to developers of promising medicines, to optimize the generation of strong data and enable accelerated evaluation so these medicines can potentially reach patients faster. . PRIME’s goal is to help patients benefit as soon as possible from innovative new therapies that have the potential to significantly address an unmet medical need. The PRIME designation was granted based on proof-of-concept clinical data from Vertex’s Phase 2 study of inaxapline in APOL1-mediated FSGS. Inaxaplin is only the second nephrology product to receive the PRIME designation.

Vertex now holds three of approximately 70 non-oncology PRIME designations granted to date, including its two PRIME designations for exagamglogene autotemcel (exa-cel), formerly known as CTX001, one for transfusion-dependent beta-thalassemia and one for sickle cell disease. In the United States, this is the ninth breakthrough therapy designation granted to Vertex across all of its portfolio programs.

About the Inaxaplin Pivotal Program (VX-147)
A phase 2/3 adaptive randomized, double-blind, placebo-controlled study is underway that will initially evaluate two doses of inaxaplin over 12 weeks to select a dose for phase 3 and then assess the efficacy and safety of the single dose selected in the phase 3 study.

Patients between the ages of 18 and 60, with two APOL1 mutations, urine protein to creatinine ratio (UPCR) ≥ 0.7 g/g to 2 and on stable doses of standard-of-care medications are eligible to enroll. It is expected to enroll approximately 66 patients in the Phase 2 dose-finding portion of the study, and an additional 400 patients are expected to be enrolled in the Phase 3 portion of the study.

The primary efficacy endpoint for the final analysis is the slope of eGFR in patients receiving the selected dose of inaxapline compared to placebo. The secondary efficacy endpoint is the time to composite clinical outcome, which will also be assessed in the final analysis and is defined as a sustained decline of ≥30% from baseline in eGFR, onset end-stage renal disease (i.e. maintenance dialysis for ≥ 28 days, renal transplantation, or sustained eGFR 2), or death. Final study analysis will occur when subjects have at least two years of eGFR data and when approximately 187 composite clinical outcomes have occurred.

The study is also designed to have a pre-planned interim analysis at week 48 assessing eGFR slope, supported by percent change from baseline in UPCR in the inaxapline arm versus the placebo. If positive, the interim analysis may serve as the basis for Vertex to seek accelerated approval of inaxapline in the United States for patients with AMKD.

About APOL1-mediated renal failure
APOL1-mediated renal failure is a form of chronic renal failure caused by mutations in the APOL1 embarrassed. About 100,000 people in the United States and Europe have two APOL1 genetic mutations and proteinuric kidney disease. People who inherit two mutations in the APOL1 gene have a disease course that is much more aggressive than in the absence of APOL1 genetic mutations. Inherited APOL1 genetic mutations cause kidney disease through a toxic gain of function, which results in damage to podocytes. This lesion disrupts filtration, leading to proteinuria and rapidly progressive kidney disease. Progressive kidney disease can lead to dialysis, kidney transplantation, or death.

About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has several approved drugs that treat the underlying cause of cystic fibrosis (CF) – a rare and life-threatening genetic disease – and has several ongoing CF clinical and research programs. Beyond cystic fibrosis, Vertex has a strong portfolio of experimental small molecule, cellular and genetic therapies in other serious diseases where it has in-depth knowledge of the causal human biology, including sickle cell disease, beta-thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes, alpha-1 antitrypsin deficiency, and Duchenne muscular dystrophy.

Founded in 1989 in Cambridge, Mass., Vertex’s global headquarters are now located in Boston’s Innovation District and its international headquarters are in London. Additionally, the company has research and development sites and sales offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the best places to work in the industry, including 12 consecutive years on Science magazine’s Top Employers list and one of Seramount’s (formerly Working Mother Media) Top 100 Companies in 2021. For company updates and to learn more about Vertex’s innovation history, visit or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Special note regarding forward-looking statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the accelerated development of inaxapline resulting from therapy designation breakthrough status and PRIME designation from the EMA, the potential benefits of inaxaplin, the anticipated timing and dosage associated with ongoing and future clinical trials, the study design, including expectations regarding the patient recruitment, expectations for efficacy endpoints, plans for interim evaluation, and plans for submission for regulatory approval in the United States. the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the Company’s beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause the actual events or results from those expressed or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, that data from a limited number of patients may not be indicative of the ultimate results of the clinical trial, that the trial may not be completed within the expected time frame, or not at all, that data from the company’s development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under “Risk Factors” in Vertex’s most recent annual report filed with the Securities and Exchange Commission (SEC) and available through the company’s website at and at SEC website at You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.