Voydeya Receives FDA Approval as Supplemental Treatment with Ravulizumab or Eculizumab for Managing Extravascular Hemolysis in Adult Patients with PNH

Voydeya (danicopan) has received approval in the United States for use alongside ravulizumab or eculizumab in treating extravascular hemolysis (EVH) in adults diagnosed with paroxysmal nocturnal hemoglobinuria (PNH). This innovative oral Factor D inhibitor, Voydeya, is the first of its kind and is designed to complement the standard treatments Ultomiris (ravulizumab) or Soliris (eculizumab). Its purpose is to cater to the needs of the roughly 10-20% of PNH patients who undergo clinically significant EVH despite being treated with a C5 inhibitor.

The FDA’s authorization was based on favorable outcomes from the crucial ALPHA Phase III study. The findings from the initial 12-week assessment phase of the trial were disclosed in The Lancet Haematology.

Marc Dunoyer, CEO of Alexion, stated that the authorization of Voydeya, the first-of-its-kind Factor D inhibitor, signifies a significant step forward in PNH treatment. This achievement underscores their dedication to pioneering advancements in complement science. According to the findings of the ALPHA trial, the combined inhibition of the complement pathways at Factor D and C5 could offer an effective treatment strategy for patients with EVH, allowing them to maintain their established standard-of-care therapies.

The ALPHA Phase III study examined how effective and safe Voydeya is when used alongside Ultomiris or Soliris in PNH patients with notable EVH. Findings revealed that Voydeya successfully achieved the main goal of altering hemoglobin levels from the beginning to the 12th week, as well as meeting all important secondary objectives, such as avoiding transfusions and altering the Functional Assessment of Chronic Illness Therapy – Fatigue (FACIT-Fatigue) score.

Voydeya, the PNH treatment drug has received Breakthrough Therapy designation from the FDA and has been granted PRIME status by the European Medicines Agency. Additionally, it has earned Orphan Drug Designation in the US, the European Union (EU), and Japan for addressing PNH. Voydeya has been officially sanctioned in Japan and is currently under consideration for approval in the EU. Regulatory assessments are underway in other nations as well.

FDA Approves Vafseo Tablets by Akebia for Treating Anemia in Adults with Chronic Kidney Disease on Dialysis

Akebia Therapeutics, Inc. has revealed that the FDA approved for Vafseo® (vadadustat) Tablets to address anemia resulting from chronic kidney disease among adults undergoing dialysis for a minimum of three months. Vafseo, an oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor taken once daily, triggers the body’s natural response to low oxygen levels to boost the internal production of erythropoietin, aiding in anemia management. This medication is now sanctioned in 37 nations.

John P. Butler, CEO of Akebia, expressed pride in providing a new treatment option for the countless Americans undergoing dialysis and dealing with anemia due to CKD, with the approval of Vafseo in the U.S. He emphasized Akebia’s dedication to kidney patients, stating that this milestone was achieved through the team’s commitment. Butler believes this dedication puts the company in a unique position to successfully launch and potentially establish a new oral standard of care for dialysis patients.

The approval of Vafseo for treating anemia in adults with CKD undergoing dialysis for over three months relies on effectiveness and safety data from the INNO2VATE program, along with an evaluation of post-marketing safety data from Japan, where Vafseo was introduced in August 2020. Akebia plans to introduce Vafseo in the U.S. using its experienced commercial team specializing in renal care and by capitalizing on its partnership with CSL Vifor, a leading company in introducing innovative therapies to U.S. dialysis facilities. Following the approved guidelines, Akebia will implement a launch strategy to promote Vafseo’s adoption as a new oral standard of care for adult dialysis patients.

Bristol Myers Squibb Shares Progress of Phase III YELLOWSTONE Trial with Oral Zeposia in Moderate to Severe Active Crohn’s Disease

Bristol Myers Squibb provided an update after reviewing the initial findings from the first of two induction studies within the Phase III YELLOWSTONE clinical trial program, which is examining Zeposia (ozanimod) in adults with moderate to severe active Crohn’s disease. The trial did not achieve its main goal of achieving clinical remission by Week 12.

The safety characteristics of Zeposia in this research aligned with what has been seen in earlier studies. A comprehensive analysis of the YELLOWSTONE trial findings will be conducted by the company, collaborating with researchers to disseminate the outcomes to the scientific community at a later date.

Roland Chen, MD, senior vice president and head of Immunology, Cardiovascular, and Neuroscience development at Bristol Myers Squibb, expressed, “Thus far, no S1P modulator has demonstrated efficacy in a Phase III trial for Crohn’s disease, an area where there is a significant need for new treatments to provide relief from symptoms and the possibility of remission for more patients. While we are disappointed that the main goal was not achieved in this initial induction trial, our dedication to advancing groundbreaking science for those with immune-mediated conditions remains unwavering. We extend our gratitude to the investigators and patients involved in the YELLOWSTONE clinical trial program.”

YELLOWSTONE is a Phase III clinical trial initiative conducted across multiple centers. It comprises two 12-week induction trials, a 52-week maintenance trial, and a 264-week open-label extension trial. YELLOWSTONE aims to assess the safety and effectiveness of orally administered Zeposia (ozanimod) compared to a placebo in patients diagnosed with Crohn’s disease. Each of the induction trials involves around 600 participants, with those who respond positively proceeding to the maintenance trial. Patients who do not respond, experience disease relapse during maintenance, or complete the maintenance trial, are given the choice to join the open-label extension study. Throughout the trial program, patients are administered Zeposia at a dosage of 0.92 mg (equivalent to 1 mg).

The main focus in the induction trials is the percentage of individuals who achieve a Crohn’s Disease Activity Index (CDAI) score below 150. In the maintenance study, the key goals include the percentage of patients with a CDAI score below 150 and the percentage of patients who experience at least a 50% reduction in their Simple Endoscopic Score for Crohn’s disease (SES-CD) compared to the baseline.

FDA Greenlights Astellas’ Supplemental New Drug Application for IZERVAY in Geographic Atrophy

Astellas Pharma Inc. has announced that the FDA has accepted the company’s supplemental New Drug Application (sNDA). This application aims to include the positive 2-year data in the U.S. Prescribing Information for IZERVAY (avacincaptad pegol intravitreal solution) for treating geographic atrophy resulting from age-related macular degeneration (AMD). The sNDA is built upon findings from the GATHER2 Phase III clinical trial, which assessed the effectiveness and safety of both monthly (EM) and every other month (EOM) dosing over two years.

According to the Prescription Drug User Fee Act (PDUFA), the FDA has established a goal date for action by November 19, 2024. This announcement comes after the U.S. Centers for Medicare and Medicaid Services introduced a permanent J-code for IZERVAY, effective April 1, 2024, with the anticipation that this will speed up patient access across the United States.

“Astellas is dedicated to introducing groundbreaking therapies for individuals affected by retinal conditions like geographic atrophy. We are satisfied with the FDA’s choice to assess our submission and eagerly anticipate collaborating with the Agency during the evaluation period.”

Carolyn Sasse, Development Head, Cell and Gene Therapy, Astellas Pharma

The GATHER2 data showed that IZERVAY consistently decreased the rate of geography atrophy lesion progression in individuals with geography atrophy caused by AMD over 2 years when compared to a placebo. The advantage of treatment with IZERVAY compared to the placebo became evident as soon as 6 months into the study, continued to improve throughout the 2 years, and more than doubled in effectiveness from the first year to the second year.

IZERVAY received FDA approval on August 4, 2023, for addressing geography atrophy associated with AMD and is presently undergoing evaluation by the European Medicines Agency.

Truqap and Faslodex Obtain MHLW Approval for Advanced HR-positive Breast Cancer Therapy

AstraZeneca’s Truqap (capivasertib) alongside Faslodex (fulvestrant) has received authorization in Japan for managing adult individuals with HR-positive, HER2-negative breast cancer that is unresectable or recurrent due to PIK3CA, AKT1, or PTEN alterations, after progression following prior endocrine therapy.

The Japanese Ministry of Health, Labour, and Welfare (MHLW) granted approval following the findings from the CAPItello-291 Phase III trial featured in The New England Journal of Medicine. In this study, the combination of Truqap and Faslodex demonstrated a 50% reduction in the risk of disease progression or mortality compared to Faslodex alone in patients with tumors exhibiting PI3K/AKT pathway biomarker alterations. The hazard ratio was 0.50 with a 95% confidence interval of 0.38-0.65 (p=<0.001), and the median progression-free survival (PFS) was 7.3 months versus 3.1 months.

“Breast cancer stands as the leading form of cancer in Japanese women, emphasizing the pressing need for fresh, advanced treatment choices. The recent authorization of Truqap, an AKT-inhibitor pioneering in its class, marks a substantial advancement in addressing HR-positive breast cancer. It introduces a crucial alternative for roughly half of the patients harboring tumors featuring these distinct mutations or changes.”

Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca

In the CAPItello-291 study, the safety characteristics of Truqap in conjunction with Faslodex resembled those seen in prior trials examining this duo. Additionally, a companion diagnostic tool has been sanctioned by the MHLW to identify the pertinent mutations (PIK3CA, AKT1, and PTEN). Presently, regulatory submissions are being assessed in China, the European Union, and various other nations. The endorsement of Truqap combined with Faslodex for this purpose has already been granted in the US and several other regions, substantiated by findings from the CAPItello-291 trial.