Kyowa Kirin, Inc.
🇯🇵Japan
- Country
- 🇯🇵Japan
- Ownership
- -
- Established
- 1949-01-01
- Employees
- 7.1K
- Market Cap
- $12B
US Orphan Drug Market Set to Exceed $190 Billion by 2030 as FDA Designations Accelerate
• The US orphan drug market is projected to surpass $190 billion by 2030, with over 7,300 molecules receiving FDA Orphan Drug Designation to date, of which approximately 17.9% have gained approval.
• Since 2020, more than half of all new drug approvals by the FDA's Center for Drug Evaluation and Research have been granted orphan status, highlighting the growing importance of rare disease treatments in pharmaceutical development.
• Despite criticism over high pricing, exemplified by Abeona Therapeutics' Zevaskyn at $3.1 million per treatment, the orphan drug model has evolved into a sound business strategy offering fewer competitors, faster approvals, and seven-year market exclusivity.
Orchard Therapeutics' Lenmeldy Shows Long-Term Efficacy in Treating Metachromatic Leukodystrophy, NEJM Study Reveals
• Lenmeldy (atidarsagene autotemcel), Orchard Therapeutics' hematopoietic stem cell gene therapy, demonstrated significant extension of life expectancy and preservation of motor and cognitive function in children with early-onset metachromatic leukodystrophy.
• The New England Journal of Medicine publication details over 12 years of follow-up data with more than 250 patient-years of experience, showing durable efficacy in pre-symptomatic and early-symptomatic patients.
• Orchard Therapeutics will present six studies at the upcoming American Society of Gene and Cell Therapy meeting, highlighting their expanding portfolio of hematopoietic stem cell gene therapies for rare neurometabolic diseases.
CAR-T Cell Therapies Drive Growth in Adult T-Cell Leukemia-Lymphoma Market Despite Access Challenges
• The global Adult T-cell Leukemia-Lymphoma (ATLL) market is projected to experience significant growth through 2035, driven by increasing HTLV-1 infections and advancements in immunotherapy technologies.
• CAR-T cell therapies from companies like Gilead Sciences, Bristol Myers Squibb, and Novartis are revolutionizing ATLL treatment, though high costs—approximately $525,000 per infusion—create substantial access barriers.
• Pharmaceutical companies are working to reduce CAR-T manufacturing turnaround time by up to 50%, potentially improving outcomes for patients with aggressive forms of ATLL where timely intervention is critical.
Kura Oncology Initiates Phase 1 Trial Combining Ziftomenib with Imatinib for Advanced GIST Treatment
• Kura Oncology has dosed first patients in KOMET-015, a Phase 1 trial evaluating ziftomenib in combination with imatinib for advanced gastrointestinal stromal tumors (GIST) after imatinib failure.
• Preclinical studies show the combination provides robust antitumor activity in both imatinib-sensitive and imatinib-resistant GIST models through a synthetic lethal mechanism targeting tumor vulnerabilities.
• With approximately 60% of GIST patients developing imatinib resistance within two years, this novel menin inhibitor combination represents a potential breakthrough for the estimated 4,000-6,000 new GIST cases diagnosed annually in the U.S.
FDA Approves Lenmeldy: $4.25 Million Gene Therapy for Rare Childhood Disease MLD
• Orchard Therapeutics' Lenmeldy (atidarsagene autotemcel) has become the first FDA-approved treatment for metachromatic leukodystrophy (MLD), a rare genetic disorder that causes rapid neurological deterioration in children.
• At $4.25 million per one-time treatment, Lenmeldy now holds the title of the most expensive medicine in the United States, slightly higher than its European price of $3.7 million where it's marketed as Libmeldy.
• Clinical trials showed remarkable results with all 37 treated children surviving to age six (compared to 58% in control groups), with 71% able to walk independently and 85% maintaining normal language and cognitive function.
Novo Nordisk Launches NovoCare® Pharmacy, Offering Authentic Wegovy® at $499 Monthly
• Novo Nordisk has introduced NovoCare® Pharmacy, providing FDA-approved Wegovy® (semaglutide) at $499 per month for cash-paying patients, with convenient home delivery service.
• The new service aims to combat the rising problem of counterfeit semaglutide products by offering reliable access to authentic medication in a once-weekly, single-dose pen format.
• This initiative addresses affordability concerns for weight management medication while ensuring patients receive legitimate FDA-approved treatment amid growing demand for GLP-1 receptor agonists.
Kyowa Kirin and La Jolla Institute Extend 35-Year Research Alliance with Focus on Cell and Gene Therapies
• The 35-year partnership between Kyowa Kirin and La Jolla Institute for Immunology has been renewed for three more years, representing one of the longest-standing industry-academic collaborations in drug discovery.
• The alliance has already produced significant advances in immunology research, including a promising immunotherapy for atopic dermatitis currently in Phase III clinical trials.
• The renewed agreement will prioritize development of cell and gene therapies for diseases with limited treatment options, with preliminary clinical data for several compounds expected in 2025.
Amgen's Uplizna Shows Sustained Efficacy in Myasthenia Gravis Patients Through One Year
• Amgen's Uplizna demonstrated durable efficacy in patients with acetylcholine receptor antibody-positive generalized myasthenia gravis, with 72.3% of treated patients showing significant improvement in daily living activities versus 45.2% on placebo at 52 weeks.
• The anti-CD19 antibody therapy maintained its efficacy with an infrequent dosing schedule of just two doses per year following an initial loading dose, potentially offering a competitive advantage over current treatments requiring more frequent administration.
• Regulatory filing for Uplizna in generalized myasthenia gravis is anticipated in the first half of 2025, with the FDA having already granted the therapy Orphan Drug Designation for this indication.
Rocatinlimab Shows Promising Results in Phase 3 Trial for Moderate to Severe Atopic Dermatitis
• Amgen and Kyowa Kirin's Phase 3 IGNITE study of rocatinlimab met all primary and secondary endpoints, with 42.3% of patients in the higher dose group achieving EASI-75 compared to placebo.
• Rocatinlimab, an investigational anti-OX40 monoclonal antibody, works as a T-cell rebalancing therapy targeting a root cause of atopic dermatitis, potentially offering a new mechanism of action for patients.
• The ROCKET clinical program includes multiple studies evaluating rocatinlimab's efficacy, with ongoing trials examining long-term maintenance, durability of response, and effectiveness in adolescent populations.
UK Faces Growing Challenges in Access to Rare Disease Medicines Despite Recent Approvals
• Recent data reveals a concerning trend as UK drops from 2nd to 10th place in European rankings for orphan medicine availability, signaling declining access to rare disease treatments.
• Analysis shows over 100% increase in terminated NICE appraisals over the past decade, with rates rising from 9.8% (2016-2019) to 20.1% (2020-2023), limiting patient access to licensed medicines.
• Industry survey indicates 11 out of 18 pharmaceutical companies expect to launch less than 75% of their rare disease pipeline in the UK, citing unfavorable reimbursement environment and high payment rates.
Fotivda-Opdivo Combination Shows Comparable Quality of Life Outcomes to Fotivda Alone in Advanced Kidney Cancer
• Phase 3 TiNivo-2 study demonstrates that combining Fotivda (tivozanib) with Opdivo (nivolumab) maintains similar patient-reported outcomes compared to Fotivda monotherapy in advanced renal cell carcinoma patients.
• Approximately 75% of patients in both treatment arms reported improved or stable kidney cancer symptoms, with consistent quality of life scores maintained throughout the study period.
• Safety profile analysis reveals lower rates of certain side effects in the combination therapy group, potentially due to lower dosing requirements, while maintaining established efficacy parameters.
Kura Oncology's Menin Inhibitor Ziftomenib Shows Promise in Mid-Stage Cancer Trial
• Kura Oncology and Kyowa Kirin report positive results from their Phase 2 clinical trial evaluating ziftomenib, a novel menin inhibitor for cancer treatment.
• Despite the encouraging trial results, Kura Oncology's stock experienced an unexpected 9% decline in regular trading, while competitor Syndax saw gains.
• The development marks a significant milestone in targeted cancer therapy, though market response suggests complex factors affecting investor confidence.
Ziftomenib Achieves Primary Endpoint in Pivotal AML Trial, Kura and Kyowa Kirin Prepare FDA Filing
• Kura Oncology and Kyowa Kirin's oral menin inhibitor ziftomenib met its primary endpoint in the KOMET-001 trial for NPM1-mutated relapsed/refractory acute myeloid leukemia patients.
• The drug demonstrated a favorable benefit-risk profile with safety and tolerability consistent with previous reports, positioning it for an FDA New Drug Application submission in Q2 2024.
• Complete trial data will be presented at an upcoming medical conference, with analysts projecting a complete response rate between 20-30% based on study parameters.
DelveInsight Report: Over 50 Pipeline Drugs in Development for Urothelial Carcinoma Treatment
• DelveInsight's latest pipeline analysis reveals 40+ pharmaceutical companies actively developing 50+ therapeutic candidates for urothelial carcinoma treatment, indicating robust research activity in this space.
• Major pharmaceutical companies including AstraZeneca, Seagen, and Janssen are advancing promising therapies, with notable candidates like disitamab vedotin and cetrelimab in late-stage development.
• Recent clinical trials include Seagen's Phase 2 study of disitamab vedotin with pembrolizumab, and Roche's Phase 1b/II umbrella study investigating multiple combination therapies for advanced urothelial carcinoma.
Galderma's Relfydess Shows Rapid, Long-Lasting Improvement for Frown Lines in Phase IIIb Trial
• Phase IIIb RELAX trial data demonstrates rapid onset of aesthetic improvement with Relfydess (RelabotulinumtoxinA) for frown lines, with 40% of subjects reporting improvement on Day 1.
• The study showed long-lasting efficacy, with 69% of subjects satisfied at Month 6 and 60% at Month 12, highlighting sustained benefits of the treatment.
• RelabotulinumtoxinA continues to be well-tolerated, with no treatment-related serious adverse events reported, reinforcing its safety profile.
• Galderma's Relfydess has received approvals in 14 European countries, Australia, and the UK, expanding its availability for aesthetic use.
Biopharma Industry Faces Continued Layoffs in 2025 Amid Strategic Realignments
• Biopharma companies continue to announce layoffs in 2025, driven by strategic realignments, pipeline reprioritizations, and challenging financial conditions.
• Several companies, including Intellia Therapeutics and IGM Biosciences, are cutting a significant portion of their workforce to focus on key programs and extend cash runways.
• These workforce reductions are often accompanied by discontinuation of certain drug development programs and a shift in strategic focus towards core assets.
• The layoffs reflect a broader trend in the industry as companies adapt to evolving market dynamics and prioritize efficiency and long-term sustainability.
BridgeBio's Attruby Receives FDA Approval for ATTR-CM Treatment
• The FDA has approved BridgeBio's Attruby (acoramidis) for treating adults with transthyretin amyloid cardiomyopathy (ATTR-CM), marking a significant milestone for the company.
• Attruby is the first and only approved product with a label specifying near-complete stabilization of transthyretin, demonstrating benefits on cardiovascular outcomes.
• Clinical trials showed Attruby significantly reduced death and cardiovascular-related hospitalization, while also improving the quality of life for patients with ATTR-CM.
• Priced at $225,000 per year, Attruby offers a new treatment option in the ATTR-CM market, though it will compete with existing therapies like Pfizer's tafamidis.
Ziftomenib Shows Promise in AML: Kura Oncology and Kyowa Kirin Advance to Phase 3 Trials
• Kura Oncology and Kyowa Kirin's ziftomenib demonstrates statistically significant efficacy in Phase 2 trial for relapsed/refractory NPM1-mutant acute myeloid leukemia (AML).
• The companies plan to submit a New Drug Application (NDA) to the FDA in the second quarter of 2025, seeking approval for ziftomenib in R/R NPM1-m AML.
• Phase 3 trials are set to begin in the second half of 2025, evaluating ziftomenib in combination with intensive and non-intensive chemotherapy regimens for newly diagnosed AML.
• FDA feedback supports potential accelerated approval pathways in both intensive and non-intensive treatment settings, based on MRD negative CR and CR endpoints.
© Copyright 2025. All Rights Reserved by MedPath