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Health Canada Extends Evkeeza Approval to Infants as Young as 6 Months for Rare Cholesterol Disorder

Drug ApprovalRare DiseaseMonoclonal Antibody
  • Health Canada has extended approval of Evkeeza (evinacumab) to treat children as young as 6 months old with homozygous familial hypercholesterolemia (HoFH), a devastating inherited cholesterol disorder.
  • HoFH affects 1 in 300,000 people globally and causes dangerously high LDL cholesterol levels exceeding 10 mmol/L, putting patients at risk for premature heart disease and cardiac events.
  • Evkeeza works by blocking the ANGPTL3 protein to reduce cholesterol levels and is administered monthly via infusion alongside low-fat diet and other cholesterol-lowering medications.
  • The therapy is now commercially available in multiple countries including Canada, the UK, US, Italy, Japan, and several European nations, with early access programs in 13 additional countries.

Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

NCT03399786CompletedPhase 3
Regeneron Pharmaceuticals
Posted 1/18/2018

Sun Pharma Launches Deuruxolitinib for Severe Alopecia Areata in US Market

Drug Approval
  • Sun Pharmaceuticals has launched deuruxolitinib (Leqselvi) in the United States for treating severe alopecia areata in adults, offering 8 mg tablets to patients aged 18 and older.
  • Clinical trials demonstrated that approximately one-third of patients achieved 80% or more scalp hair coverage by week 24, with some reaching 90% coverage from an initial average of only 13%.
  • Long-term data showed sustained efficacy with 48.8% of patients achieving SALT scores ≤20 by week 68, and 99.6% of initial responders maintaining their response.
  • The company has introduced the Leqselvi Support Program offering eligible patients medication for as little as $0 for up to two years with personalized assistance.

Study to Evaluate the Efficacy and Safety of CTP-543 in Adults With Moderate to Severe Alopecia Areata (THRIVE-AA1)

NCT04518995CompletedPhase 3
Concert Pharmaceuticals
Posted 11/23/2020

Study to Evaluate the Efficacy and Safety of CTP-543 in Adults With Moderate to Severe Alopecia Areata (THRIVE-AA2)

NCT04797650CompletedPhase 3
Concert Pharmaceuticals
Posted 6/10/2021

Endometriosis Pain Treatment Pipeline Shows Mixed Results as NHS Approves First Daily Oral Therapy

Monoclonal AntibodyDrug Approval
  • The NHS in England has approved relugolix combination therapy as the first long-term daily oral treatment for endometriosis, expected to benefit around 1,000 women annually whose symptoms persist despite previous treatments.
  • Organon discontinued development of OG-6219 after the drug candidate failed its Phase 2 trial in July 2025, despite being acquired for $75 million and previously described as the company's "biggest potential opportunity."
  • Hope Medicine's HMI-115, a first-in-class monoclonal antibody targeting the prolactin receptor, reported positive interim Phase II results and received Breakthrough Therapy Designation from China's NMPA.
  • Multiple companies including TiumBio, Lisata Therapeutics, and Gynica are advancing novel therapeutic approaches through various clinical trial phases, representing diverse mechanisms of action for endometriosis pain management.

A Study of E7386 in Combination With Other Anticancer Drug(s) in Participants With Solid Tumor

NCT04008797RecruitingPhase 1
Eisai Inc.
Posted 7/11/2019

Ascentage Pharma Raises $192M in Oversubscribed Share Placement to Advance Cancer Pipeline

Drug Approval
  • Ascentage Pharma completed a $192.3 million share placement that was oversubscribed by eight times, demonstrating strong investor confidence in the company's cancer-focused pipeline.
  • The proceeds will fund commercialization efforts for approved drugs olverembatinib and lisaftoclax, expand global clinical development, and strengthen operations infrastructure.
  • The company is advancing multiple Phase III trials including POLARIS-2 for olverembatinib in CML and four GLORA studies for lisaftoclax across various hematologic malignancies.
  • Lisaftoclax recently received NMPA approval in China for relapsed/refractory CLL and SLL, while olverembatinib is already approved and covered by China's National Reimbursement Drug List.

Indian Regulators Reject Troikaa's Rapid IV Paracetamol Bolus Over Safety and Efficacy Concerns

Drug Approval
  • India's Subject Expert Committee (SEC) has rejected Troikaa Pharmaceuticals' proposal for a rapid intravenous paracetamol bolus formulation due to insufficient safety and efficacy data.
  • The proposed formulation would deliver paracetamol 1000 mg/4 ml as a 2-minute IV bolus, significantly faster than the standard 15-minute infusion currently used.
  • Regulators flagged the absence of commonly expected adverse events in Phase III trial data and questioned the clinical support for proposed indications.
  • The committee has requested additional studies and data before reconsidering approval for the concentrated paracetamol formulation.

Darolutamide Gains FDA Approval for Metastatic Hormone-Sensitive Prostate Cancer Following ARANOTE Trial Success

Drug Approval
  • Darolutamide (Nubeqa) received FDA approval on June 3, 2025, for treating metastatic hormone-sensitive prostate cancer based on the ARANOTE trial results.
  • The ARANOTE trial demonstrated efficacy of darolutamide plus androgen deprivation therapy as a doublet regimen, expanding treatment options beyond existing triplet therapies.
  • NCCN guidelines now include four Category 1 preferred oral agents for mHSPC: abiraterone, apalutamide, enzalutamide, with darolutamide expected to be upgraded from Category 2B status.
  • The approval provides patients with low-volume metachronous disease an additional oral treatment option that minimizes adverse events while maintaining aggressive therapeutic approach.

Targeted Protein Degradation Emerges as Multi-Billion Dollar Therapeutic Frontier with First PROTAC Approval Expected in 2026

Drug ApprovalOncology
  • Targeted protein degradation (TPD) has evolved from niche science to mainstream drug development, with over 40 PROTAC candidates in clinical testing and the first potential market approval expected for Arvinas/Pfizer's ARV-471 by June 2026.
  • Major pharmaceutical companies have committed over $10 billion in partnerships since 2024, with deals including AbbVie's $1.64 billion agreement with Neomorph and LEO Pharma's $1.7 billion alliance with Gilead Sciences.
  • The Asia-Pacific region, particularly China and South Korea, has emerged as a leading hub for TPD research, with multiple companies advancing candidates through late-stage clinical trials.
  • The global TPD market is projected to surge from $1 billion currently to $6.94 billion by 2035, driven by advances in computational tools and AI-enabled drug design.

FDA Approves Retifanlimab Plus Carboplatin/Paclitaxel for Advanced Anal Cancer Following PODIUM-303 Trial Success

Drug Approval
  • The FDA approved retifanlimab in combination with carboplatin/paclitaxel for advanced anal cancer in May 2025, based on the PODIUM-303 study results.
  • The PODIUM-303 trial demonstrated improved progression-free survival (9.3 vs 7.4 months) and response rates (56% vs 44%) with the addition of retifanlimab to standard chemotherapy.
  • Current NCCN guidelines now include carboplatin/paclitaxel plus retifanlimab as a category 2B evidence-based approach for first-line treatment of advanced squamous cell anal cancer.
  • The InterAACT study previously established carboplatin/paclitaxel as standard care, showing similar response rates but improved survival and tolerability compared to cisplatin/5-FU.

Soleno Therapeutics Raises $200 Million to Fund Commercialization of First FDA-Approved Prader-Willi Syndrome Therapy

Drug ApprovalRare Disease
  • Soleno Therapeutics completed a $200 million public offering at $85 per share to fund commercialization of VYKAT XR, the first FDA-approved therapy for hyperphagia in Prader-Willi syndrome patients.
  • The company received FDA approval for VYKAT XR on March 26, 2025, marking a significant milestone for treating this rare genetic disorder.
  • Proceeds will also support regulatory and market development activities in the European Union and further research and development efforts.
  • The offering included 2,352,941 shares with underwriters holding a 30-day option to purchase an additional 352,941 shares at the same price.

FDA Approves First Year-Long Flea and Tick Injectable for Dogs

Drug Approval
  • The FDA has approved BRAVECTO QUANTUM, the first and only parasiticide that protects dogs against fleas and ticks for an entire year with a single injection.
  • The injectable suspension contains fluralaner and is indicated for dogs and puppies 6 months of age and older, with availability expected at veterinary clinics nationwide by August 2025.
  • BRAVECTO QUANTUM provides 12-month protection against most tick species and fleas, though protection against lone star ticks is limited to 8 months.
  • The product has already been approved in more than 50 countries worldwide, including Australia, New Zealand, and the European Union prior to U.S. approval.

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