Larotrectinib

US Orphan Drug Market Set to Exceed $190 Billion by 2030 as FDA Designations Accelerate

9 days ago

• 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.

FDA Phases Out Animal Testing Requirement for Monoclonal Antibodies and Other Drugs

a month ago

• FDA has announced plans to eliminate mandatory animal testing requirements for monoclonal antibodies and certain other pharmaceutical products, marking a significant shift in drug development regulations. • The announcement comes amid broader regulatory changes at FDA, including recent approvals of cancer treatments such as nivolumab with ipilimumab for hepatocellular carcinoma and durvalumab for muscle invasive bladder cancer. • HHS Secretary Robert F. Kennedy Jr. framed these changes as part of a "generational opportunity" to reform the regulatory agency, while the department undergoes significant restructuring with personnel cuts and division consolidations.

Bayer's Vitrakvi Makes History as First Tumor-Agnostic Drug Approved in European Union

3 months ago

• The European Medicines Agency (EMA) has approved Vitrakvi (larotrectinib) as the first-ever tumor-agnostic therapy, treating cancers based on molecular signatures rather than tumor location. • The drug targets rare NTRK gene fusion cancers affecting several thousand European patients annually, particularly prevalent in sarcomas, brain, kidney, and thyroid cancers. • Bayer's Vitrakvi leads the European market ahead of competitor Roche's Rozlytrek, with approvals already secured in the US, Brazil, and Canada.

Verastem Oncology to Present New LGSOC Treatment Data at SGO 2025 Annual Meeting

3 months ago

• Verastem Oncology will present additional analyses from the Phase 2 RAMP 201 trial evaluating avutometinib plus defactinib in recurrent low-grade serous ovarian cancer at SGO 2025, building on their FDA Priority Review submission. • The presentations include new subgroup analyses by KRAS mutational status, supporting the company's ongoing efforts to advance treatment options for RAS/MAPK pathway-driven cancers. • The company's NDA for the avutometinib-defactinib combination therapy has received Priority Review from the FDA with a PDUFA date of June 30, 2025, for KRAS mutant LGSOC patients.

FDA Approves Lilly's Retevmo as First Targeted Therapy for RET-Positive Lung and Thyroid Cancers

3 months ago

• Eli Lilly's Retevmo (selpercatinib) receives FDA approval as the first targeted therapy for RET-altered lung and thyroid cancers, demonstrating response rates up to 84% in clinical trials. • The drug showed remarkable efficacy with 64-84% overall response rates in non-small cell lung cancer and 69-73% response rates in medullary thyroid cancer patients, with benefits lasting six months or longer. • Lilly plans immediate launch with pricing at $20,600 for a 30-day supply, targeting approximately 2% of NSCLC patients and up to 70% of medullary thyroid cancer patients with RET mutations.

Experts Address Key Challenges in Implementation of Tumor-Agnostic Therapies and Genetic Testing

4 months ago

• The increasing adoption of tumor-agnostic therapies has highlighted the need for universal genetic testing in oncology practices, with eight drugs now holding FDA pan-tumor approvals as of January 2025. • Healthcare providers face complex decisions regarding genetic testing methodologies, including choices between tissue and liquid biopsies, timing of collection, and interpretation of results. • Clinical implementation challenges include establishing efficient testing workflows, securing genetic counseling resources, and determining appropriate treatment sequencing in cases with limited tumor-specific data.

FDA Approves Subcutaneous Opdivo Qvantig for Multiple Solid Tumors

5 months ago

• The FDA approved Opdivo Qvantig, a subcutaneous injection of nivolumab, for adult solid tumors previously approved for intravenous Opdivo. • CheckMate-67T trial data showed non-inferior pharmacokinetic exposures, similar efficacy, and comparable safety to intravenous Opdivo. • Opdivo Qvantig offers a faster 3-5 minute administration time, providing convenience and flexibility for patients. • The approval helps Bristol Myers Squibb maintain market share amid patent expirations and increasing biosimilar competition.

Nuvation Bio's Taletrectinib Gains FDA Priority Review and Expanded Access Program for ROS1+ NSCLC

5 months ago

• The FDA granted priority review to Nuvation Bio's NDA for taletrectinib, a next-generation ROS1 inhibitor, for advanced ROS1+ non-small cell lung cancer, setting a PDUFA date of June 23, 2025. • Taletrectinib demonstrated promising efficacy and tolerability in the TRUST-I and TRUST-II trials, showing durable responses and prolonged progression-free survival in ROS1+ NSCLC patients. • Nuvation Bio has initiated an Expanded Access Program (EAP) in the U.S. for taletrectinib, offering access to eligible patients with advanced ROS1+ NSCLC outside of clinical trials. • The NDA is based on pooled data from the TRUST-I and TRUST-II studies, which included over 300 patients, representing the largest ROS1-positive NSCLC dataset supporting an NDA.

Larotrectinib Shows High Efficacy in Pediatric Solid Tumors with NTRK Fusion

6 months ago

• Larotrectinib demonstrates high effectiveness in pediatric patients with newly diagnosed infantile fibrosarcoma (IFS) and other solid tumors harboring NTRK gene fusions. • The study marks the first Children's Oncology Group (COG) trial to assess precision medicine in a front-line setting based on genetic biomarkers. • Results indicate larotrectinib could replace chemotherapy as a first-line treatment for NTRK fusion-positive tumors, reducing potential harm to developing organs. • The objective response rate within six cycles was 94% for IFS and 60% for other solid tumors, highlighting the drug's potential in pediatric cancer care.

FDA Approves Illumina's TruSight Oncology Comprehensive Assay as Pan-Tumor Companion Diagnostic

8 months ago

• The FDA has approved Illumina's TruSight Oncology (TSO) Comprehensive Assay as a pan-tumor companion diagnostic, enhancing precision medicine. • TSO Comprehensive is the first FDA-approved kit for identifying NTRK-positive solid tumor patients eligible for larotrectinib and RET fusion-positive NSCLC patients for selpercatinib. • The assay profiles over 500 biomarkers using next-generation sequencing, facilitating personalized therapy recommendations and clinical trial enrollment. • Illumina is partnering with pharmaceutical companies to expand TSO Comprehensive's companion diagnostic claims, with future claims pending regulatory approvals.

Drug Development Updates