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FDA Approves Enhanced Labeling for Endari Based on Post-Marketing Pharmacokinetic Data

Drug ApprovalRare Disease
  • Emmaus Life Sciences received FDA approval for enhanced labeling of Endari (L-glutamine oral powder) based on additional post-marketing pharmacokinetic study data.
  • The updated label provides more comprehensive prescribing information, including confirmation of body weight-based dosing, no unwanted accumulation with twice-daily dosing, and flexible administration with or without food.
  • Endari remains approved to reduce acute complications of sickle cell disease in adult and pediatric patients five years and older, addressing a condition affecting approximately 100,000 people in the United States.
  • The label enhancement represents a significant improvement in clinical guidance for healthcare providers treating sickle cell disease patients with this prescription-grade L-glutamine therapy.

Hong Kong Establishes Independent Drug Regulatory Authority by End 2026, Ending Reliance on Foreign Approvals

Drug Approval
  • Hong Kong will launch its own drug and medical device regulator by late 2026, marking a significant shift from the current secondary evaluation system that requires approval from at least two overseas regulators.
  • The Hong Kong Centre for Medical Products Regulation will implement primary evaluation in phases starting 2026, enabling fast-track approval of homegrown drugs and medical devices based on clinical trial data without foreign regulatory dependence.
  • Since 2023, 11 life-saving drugs including cancer treatments have been approved under the interim "1+" mechanism requiring only one overseas regulator's approval, demonstrating progress toward regulatory independence.
  • The city plans to apply for membership in the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use in 2027 to achieve international recognition of its regulatory framework.

Teva Partners with Formycon to Commercialize Stelara Biosimilar Fymskina in Germany

Monoclonal AntibodyDrug Approval
  • Formycon AG has entered into a distribution agreement with Teva's subsidiary Ratiopharm for the semi-exclusive commercialization of its Stelara biosimilar FYB202, branded as Fymskina, in Germany.
  • The market launch for Fymskina is scheduled for the third quarter of 2025, with Formycon handling manufacturing and supply while Ratiopharm manages commercialization.
  • Fymskina has already received European Commission approval for treating moderate to severely active Crohn's disease, moderate to severe plaque psoriasis, and active psoriatic arthritis.
  • This partnership represents Formycon's strategic approach to expand market coverage through semi-exclusive partnerships, building on its existing global commercialization agreement with Fresenius Kabi.

FDA-Approved Liquid Imatinib Formulation Addresses Critical Dosing Challenges in Pediatric Cancer and Dysphagia Patients

Drug Approval
  • The FDA approved a liquid formulation of imatinib (Imkeldi) in November 2024, providing a stable, flavored, non-refrigerated solution for pediatric cancer patients and those with dysphagia who previously faced significant barriers to safe medication administration.
  • Prior to this approval, patients requiring imatinib had to rely on tablet crushing, compounding pharmacies, or extemporaneous preparations, creating safety concerns, dosing inconsistencies, and additional burden for families managing cancer treatment at home.
  • Clinical pharmacists emphasize that liquid formulations improve treatment adherence, reduce human error in dosing, and eliminate the need for complex manipulation procedures that require protective equipment and specialized training.
  • The new formulation addresses critical gaps in pediatric-friendly oncology medications, where many oral chemotherapy agents lack appropriate dosage forms for children who cannot swallow tablets or adults with swallowing difficulties.

Oral Sulopenem Shows Non-Inferiority to Standard Care in Phase 3 Trial for Uncomplicated UTIs

Drug Approval
  • Oral sulopenem (ORLYNVAH) demonstrated non-inferiority to amoxicillin/clavulanate in treating uncomplicated urinary tract infections in adult women, with overall success rates of 60.9% versus 55.6% respectively in the phase 3 REASSURE trial.
  • The study enrolled 2,222 adult women and showed sulopenem's statistical superiority in patients with baseline uropathogens susceptible to amoxicillin/clavulanate, achieving 61.7% success compared to 55.0%.
  • Adverse events were more frequent with sulopenem, particularly diarrhea (8.1% vs 4.1%), nausea (4.3% vs 2.9%), and headache (2.2% vs 1.5%), though these were predominantly mild in severity.
  • The FDA approved oral sulopenem in October 2024 as the first oral penem antibiotic in the US, specifically for patients with limited or no alternative oral antibacterial options.

Oral Sulopenem-etzadroxil/Probenecid Versus Ciprofloxacin for Uncomplicated Urinary Tract Infection in Adult Women

NCT03354598CompletedPhase 3
Iterum Therapeutics, International Limited
Posted 8/1/2018

Oral Sulopenem Versus Amoxicillin/Clavulanate for Uncomplicated Urinary Tract Infection in Adult Women

NCT05584657CompletedPhase 3
Iterum Therapeutics, International Limited
Posted 10/18/2022

Valorum Biologics Secures Exclusive Rights to Commercialize Formycon's Eylea Biosimilar in North America

Drug Approval
  • Valorum Biologics LLC has secured exclusive commercialization rights for FYB203/AHZANTIVE®, Formycon's biosimilar to Eylea®, in the United States and Canada through a licensing agreement with Klinge Biopharma GmbH.
  • The FDA-approved biosimilar treats serious retinal diseases including wet age-related macular degeneration, diabetic macular edema, diabetic retinopathy, and macular edema following retinal vein occlusion by inhibiting VEGF.
  • Formycon will participate in mid-single-digit to low-double-digit percentage range of all payment streams and manage the supply chain, while Canadian regulatory approval is expected by the end of 2024.
  • The partnership leverages Valorum's experienced management team with proven track records at major pharmaceutical companies including Johnson & Johnson, Merck, and Roche to maximize market penetration.

Liquidia Secures $50 Million Funding Following YUTREPIA Commercial Launch and Legal Victory

Drug ApprovalRare Disease
  • Liquidia Corporation received $50 million from Healthcare Royalty following the first commercial sale of YUTREPIA and a favorable court ruling denying United Therapeutics' injunction request.
  • The company has now accessed $175 million of $200 million in total potential funding, with an additional $25 million available if YUTREPIA achieves net sales exceeding $100 million by June 2026.
  • YUTREPIA is an inhaled dry-powder formulation of treprostinil approved for treating pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease.
  • The funding will accelerate launch execution, advance clinical pipeline development, and support manufacturing facility expansion as Liquidia targets profitability without additional capital needs.

Investigation of the Safety and Pharmacology of Dry Powder Inhalation of Treprostinil

NCT03399604CompletedPhase 3
Liquidia Technologies, Inc.
Posted 1/2/2018

An Open-Label ProSpective MultiCENTer Study to Evaluate Safety and Tolerability of Dry Powder Inhaled Treprostinil in PH

NCT06129240Recruiting
Liquidia Technologies, Inc.
Posted 12/28/2023

Breckenridge Pharmaceutical Receives FDA Approval for Generic Dehydrated Alcohol Injection for Hypertrophic Obstructive Cardiomyopathy

Drug ApprovalRare Disease
  • Breckenridge Pharmaceutical received FDA approval for its generic version of Ablysinol, a dehydrated alcohol injection used to treat symptomatic hypertrophic obstructive cardiomyopathy in patients unsuitable for surgical myectomy.
  • The sterile, preservative-free solution contains ≥99% ethyl alcohol and is indicated for inducing controlled cardiac septal infarction to improve exercise capacity in adult HOCM patients.
  • This approval positions Breckenridge as one of the first companies to offer a generic alternative to Ablysinol in the U.S. market, supporting the company's strategy to expand its institutional healthcare presence.
  • The product will be available primarily in hospitals, clinics, and home health care facilities, reflecting Breckenridge's commitment to providing affordable treatment options for rare disease patients.

JB Chemicals Secures FDA Approval for Generic Amitriptyline Depression Treatment

Drug ApprovalFDA Approval
  • JB Chemicals and Pharmaceuticals Ltd received final FDA approval for its generic Amitriptyline Hydrochloride tablets in six dosage strengths for treating depression symptoms.
  • The approval allows the Indian pharmaceutical company to market its generic version of the tricyclic antidepressant in the US market, strengthening its regulated market presence.
  • This development supports JB Chemicals' international expansion strategy, with the company reporting 15.5% year-on-year net profit growth to ₹145.7 crore in the March quarter.
  • Analyst sentiment remains positive with 14 out of 15 analysts maintaining 'buy' ratings and price targets suggesting 11.4% potential upside.

Glenmark Launches Tevimbra, First Immuno-Oncology Drug in India for Lung and Esophageal Cancer Treatment

Monoclonal AntibodyDrug ApprovalOncology
  • Glenmark Pharmaceuticals has launched Tevimbra (tislelizumab), marking the company's first entry into immuno-oncology in India following CDSCO approval.
  • The anti-PD-1 monoclonal antibody is indicated for first-line treatment of locally advanced or metastatic NSCLC in combination with chemotherapy and second-line treatment as monotherapy.
  • Tevimbra is already approved in 46 countries including the US and EU, with over 1.5 million patients treated globally and worldwide sales of $625 million in 2024.
  • The drug targets NSCLC, which represents over 80% of lung cancer cases, and ESCC, the most common esophageal cancer subtype in India.

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