United Therapeutics

ISPE Celebrates 20th Anniversary with Announcement of 2025 Facility of the Year Awards Winners

8 days ago

• Six pharmaceutical facilities received category awards for innovation, operations, and social impact at the 2025 ISPE Europe Annual Conference, marking the 20th anniversary of the prestigious FOYA program. • Touchlight's synthetic DNA manufacturing facility and Johnson & Johnson's flexible packaging operations were among the winners recognized for advancing pharmaceutical manufacturing standards and sustainability initiatives. • The overall 2025 FOYA winner will be announced at a special black-tie celebration during the ISPE Annual Meeting & Expo in Charlotte, North Carolina in October 2025.

AstraZeneca Opens $300 Million Cell Therapy Manufacturing Facility in Rockville, Maryland

13 days ago

• AstraZeneca has inaugurated a new $300 million cell therapy manufacturing facility in Rockville, Maryland, designed to produce CAR-T cell therapies for cancer treatment and create over 150 specialized jobs. • The 84,000-square-foot facility is part of AstraZeneca's broader $3.5 billion investment in U.S. research and manufacturing, strengthening Montgomery County's position as a leading bio-life sciences hub. • The project received combined support of $600,000 from Montgomery County and Maryland state agencies, along with job creation tax credits and expedited permitting to accelerate the facility's completion.

Court Dismisses United Therapeutics' Challenge Against Liquidia's YUTREPIA, Clearing Path for May Launch

20 days ago

• U.S. District Court Judge Kelly dismissed United Therapeutics' cross-claim challenging Liquidia's NDA amendment for YUTREPIA, ruling that UTHR failed to establish standing. • The FDA can now grant final approval for YUTREPIA (treprostinil) inhalation powder for both PAH and PH-ILD indications after regulatory exclusivity expires on May 23, 2025. • Liquidia remains on track for a potential commercial launch with a PDUFA goal date of May 24, 2025, positioning YUTREPIA to potentially become a first-choice prostacyclin therapy for pulmonary hypertension patients.

Liquidia Secures $100M Financing to Advance Yutrepia for Pulmonary Hypertension Treatment

2 months ago

• Liquidia Corporation has amended its agreement with Healthcare Royalty to potentially add up to $100 million in financing, with an initial $25 million available at closing. • The funds will support Yutrepia's commercial development for pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD), along with clinical trials for pediatric patients. • Additional funding tranches include $50 million upon first commercial sale following FDA approval and $25 million if Yutrepia achieves over $100 million in net sales by June 2026.

Pipeline for Pulmonary Arterial Hypertension Treatment Expands with 55+ Companies Developing Novel Therapies

3 months ago

• DelveInsight's latest report reveals over 55 companies actively developing 55+ pipeline drugs for Pulmonary Arterial Hypertension treatment, indicating robust research activity in this therapeutic area. • Breakthrough therapies like Sotatercept by Acceleron Pharma and LIQ861 by Liquidia Technologies are advancing through clinical trials, offering new hope for PAH patients. • Multiple clinical trials are underway in early 2025, including studies by Actelion, Keros Therapeutics, Insmed Incorporated, and Merck Sharp & Dohme, focusing on novel treatment approaches.

Tegoprubart Successfully Used in Second Pig-to-Human Kidney Xenotransplant at MGH

3 months ago

• Massachusetts General Hospital successfully performed its second pig-to-human kidney transplant using Eledon's tegoprubart as a key immunosuppressant, with the patient now off dialysis after two years. • Tegoprubart, an anti-CD40L antibody, demonstrates potential in preventing organ rejection by blocking multiple costimulatory receptors, marking a significant advancement in xenotransplantation. • The procedure, performed on January 25, 2025, represents a crucial milestone in addressing the global organ shortage crisis, with MGH planning two additional xenotransplants this year.

United Therapeutics Completes Enrollment in Phase 3 TETON 1 IPF Trial of Inhaled Treprostinil

4 months ago

• United Therapeutics has fully enrolled its Phase 3 TETON 1 study, evaluating nebulized Tyvaso (treprostinil) for idiopathic pulmonary fibrosis (IPF). • The TETON 1 trial, with 598 patients across the U.S. and Canada, is part of a larger program assessing inhaled treprostinil in IPF and progressive pulmonary fibrosis (PPF). • Top-line data from the TETON 1 study is anticipated in the first half of 2026, potentially leading to a new treatment option for IPF patients. • If successful, United Therapeutics plans to seek FDA approval to expand Tyvaso's label to include IPF, and pursue European marketing authorization.

FDA Approves Clinical Trials for Pig Kidney Transplants in Humans, Addressing Organ Shortage

4 months ago

• The FDA has approved clinical trials for two companies, United Therapeutics and eGenesis, to transplant genetically modified pig kidneys into humans with kidney failure. • United Therapeutics will begin with six patients in mid-2025, potentially expanding to 50, while eGenesis will start with three patients, monitoring them closely. • These trials aim to address the critical shortage of donor kidneys, with over 550,000 Americans facing kidney failure and approximately 100,000 on the transplant waiting list. • Both companies have genetically modified pig genes to reduce organ rejection and improve compatibility with human bodies, but ethical and safety concerns persist.

United Therapeutics' UKidney Xenotransplant Trial Receives FDA Clearance

4 months ago

• United Therapeutics received FDA clearance for its IND application to begin clinical trials for UKidney, a gene-edited pig kidney, marking a significant step in xenotransplantation. • The phase 1/2/3 trial will enroll ESRD patients, including those ineligible for traditional transplants and those unlikely to receive a kidney within five years. • UKidney features 10 gene edits to enhance compatibility and reduce rejection, offering a potential alternative to dialysis for patients with limited options. • The trial's primary endpoints include patient and UKidney survival rates, changes in glomerular filtration rate, and quality of life improvements over a 24-week period.

Abeona Therapeutics' Pz-cel BLA Resubmission Accepted by FDA for RDEB Treatment

4 months ago

• The FDA has accepted Abeona Therapeutics' resubmitted Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) to treat recessive dystrophic epidermolysis bullosa (RDEB). • The FDA has set a PDUFA target action date of April 29, 2025, for its decision on pz-cel, an autologous cell-based gene therapy. • Abeona Therapeutics has made significant strides toward the potential commercialization of pz-cel in 2025, including discussions with payers and target treatment centers.

Genetically Engineered Pig Heart Transplants Show Promise and Challenges in Xenotransplantation

5 months ago

• Researchers report successful transplantation of 10-gene-edited pig hearts into baboons, achieving survival up to 225 days, demonstrating the potential for xenotransplantation. • The study identifies challenges related to the knockout of the CMAH gene in non-human primates, suggesting it may not be suitable for preclinical models. • Analysis of a second human xenotransplant recipient reveals insights into antibody-mediated rejection, emphasizing the need for aggressive immunosuppression strategies. • The research underscores the potential of xenotransplantation to address organ shortages while highlighting the importance of continued research to overcome immunological barriers.

Xenotransplantation Advances: Pig Kidney Transplants Show Promise in Addressing Organ Shortage

5 months ago

• Towana Looney became the longest-living recipient of a pig kidney transplant, exceeding 60 days post-surgery, marking a significant milestone in xenotransplantation. • Tim Andrews received a genetically-edited pig kidney at Massachusetts General Hospital, becoming the second living person with such a transplant and is now dialysis-free. • United Therapeutics received FDA approval for a clinical trial of its UKidney, a gene-edited porcine kidney, signaling a move towards formal xenotransplantation studies. • Researchers are sharing data and refining techniques to improve the safety and efficacy of pig organ transplants, addressing the critical shortage of human donor organs.

FDA Approves Ozempic for Reducing Kidney Disease Risks in Type 2 Diabetes Patients

5 months ago

• The FDA has approved Ozempic to reduce the risk of kidney disease worsening, kidney failure, and cardiovascular death in adults with type 2 diabetes and chronic kidney disease. • A Phase 3 clinical trial demonstrated a 24% reduction in the risk of kidney disease progression and cardiovascular death compared to placebo with Ozempic. • Ozempic is now the most broadly indicated GLP-1 receptor agonist, offering a new treatment option for millions of adults with type 2 diabetes and chronic kidney disease. • The approval was based on the FLOW trial, which showed significant benefits in kidney and cardiovascular outcomes, leading to early termination due to efficacy.

Gene Therapy Advances: From FDA Clearances to Clinical Trial Progress in Early 2025

6 months ago

• The FDA cleared United Therapeutics' UKidney for US trials, a gene-edited porcine kidney product intended for end-stage renal disease via xenotransplant. • uniQure received the green light to proceed with the second dose cohort in their Phase 1/2 trial of AMT-162 for SOD1-ALS gene therapy. • Sarepta Therapeutics' Elevidys showed sustained benefit in ambulatory patients with Duchenne muscular dystrophy in Phase 3 trial results.