Tachypnea

ROSWELL, Ga, DUBLIN and HAMILTON, Bermuda, Jan. 28, 2025 /PRNewswire/ -- Saol Therapeutics, a privately held, clinical-stage pharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for SL1009, Sodium Dichloroacetate Oral Solution (DCA). DCA will be used with a proprietary genetic test to treat an orphan pediatric mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD)1. The FDA granted Priority Review for the NDA and has set a goal date of May 27, 2025, under the Prescription Drug User Fee Act (PDUFA). Priority review is designated to applications for drugs that, if approved, would provide a significant improvement in safety or effectiveness of the treatment, prevention or diagnosis of a serious medical condition. SL1009 has previously been granted Orphan Drug and Fast Track Designations. SL1009 has also been granted a Rare Pediatric Disease Designation by the FDA and thus is eligible for a Priority Review Voucher (PRV). PDCD is a rare and life-threatening mitochondrial disorder of carbohydrate oxidation that mostly affects the nervous system and skeletal muscle and leads to decreased ATP production and energy failure. DCA is a targeted therapy that inhibits Pyruvate Dehydrogenase Kinase (PDK) to stimulate residual Pyruvate Dehydrogenase Complex (PDC) activity and increase energy (ATP) production by mitochondria. "This NDA acceptance brings us one step closer to addressing the critical health challenges faced by these children and their families, where no approved treatment is currently available," said Dave Penake, Chief Executive Officer of Saol Therapeutics. "Reaching this regulatory milestone is both a significant scientific achievement and a deeply meaningful moment for everyone who has dedicated themselves to advancing this therapy. Further, designating our application for priority review reaffirms that the FDA views PDCD as a serious condition" The NDA is supported by results from a Phase 3 double-blind placebo-controlled cross-over study (SL1009-01) and a survival study (SL1009-02). The totality of evidence submitted for review in the NDA includes mechanistic characterization, along with nonclinical and clinical evidence to demonstrate the safety and clinical benefit of DCA in PDCD patients. DCA is not currently approved for any indication in the United States. About Pyruvate Dehydrogenase Complex Deficiency (PDCD) PDCD is a mitochondrial disorder of carbohydrate oxidation that mostly affects the nervous system and skeletal muscle and leads to decreased ATP production and energy failure. It is estimated that 300-500 patients are treated in expert centers in the US, and the overall prevalence is thought to be as high as 2,000. PDCD is the most common cause of congenital lactic acidosis, a life-threatening condition that may occur as early as the neonatal period. Patients suffering from PDCD may also exhibit extreme tiredness (lethargy), poor feeding, rapid breathing (tachypnea), and other signs of neurological and neuromuscular dysfunction such as developmental delay, low muscle tone (hypotonia), abnormal eye movements and seizures. Signs and symptoms usually begin soon after birth but may appear later in childhood2. There are currently no FDA-approved therapies for PDCD. About Saol Therapeutics Saol Therapeutics (pronounced "Sail") is a privately held, clinical-stage, pharmaceutical company with operations in Roswell, GA, Dublin, Ireland and Hamilton, Bermuda. Saol is focused on development activity in CNS disorders such as spasticity and pain management, and orphan diseases. Saol is committed to providing and advancing therapeutic options for patients and the physicians treating these populations. For more information, visit . Saol Therapeutics Contact Brian Nappi, Senior Vice President, Strategy [email protected] References 1. , Accessed January 2025 2. Ganetzky R, McCormick EM, Falk MJ. Primary Pyruvate Dehydrogenase Complex Deficiency Overview. 2021 Jun 17. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Bean LJH, Gripp KW, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2023. PMID: 34138529. SOURCE Saol Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

SILVER SPRING, Md., Dec. 18, 2024 /PRNewswire/ -- Today, the U.S. Food and Drug Administration approved Ryoncil (remestemcel-L-rknd), an allogeneic (donor) bone marrow-derived mesenchymal stromal cell (MSC) therapy indicated for the treatment of steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients 2 months of age and older. Ryoncil is the first FDA-approved MSC therapy. It contains MSCs, which are a type of cell that can have various roles in the body and can differentiate into multiple other types of cells. These MSCs are isolated from the bone marrow of healthy adult human donors. "Today's decision marks an important milestone in the use of innovative cell-based therapies to treat life-threatening diseases with devastating impacts on patients, including children," said Peter Marks, M.D., Ph.D., director of the FDA's Center for Biologics Evaluation and Research (CBER). "This first mesenchymal stromal cell therapy approval demonstrates the FDA's commitment to supporting the development of safe and effective products that could improve the quality of life for patients with symptoms that are unresponsive to other therapies." Steroid-refractory acute graft-versus-host disease is a serious and life-threatening condition that can occur as a complication of allogeneic hematopoietic (blood) stem cell transplantation (allo-HSCT). In allo-HSCT, a patient receives hematopoietic stem cells from a healthy donor to replace their own stem cells and form new blood cells, a procedure often done as part of treatment for certain types of blood cancers, blood disorders or immune system disorders. "Steroid-refractory acute graft-versus-host disease can have significant, wide-ranging health consequences, including damage to multiple organs, reduced quality of life and risk of death in affected patients," said Nicole Verdun, M.D., director of the Office of Therapeutic Products in CBER. "The FDA remains dedicated to helping address the urgent unmet needs of individuals with debilitating and deadly diseases, and today's approval is an important step in that effort." The safety and effectiveness of Ryoncil were evaluated in a multicenter, single-arm study in 54 pediatric study participants with SR-aGVHD after undergoing allo-HSCT. Study participants received intravenous infusion of Ryoncil twice weekly for four consecutive weeks, for a total of eight infusions. Each study participant's condition at baseline was analyzed using the International Blood and Marrow Transplantation Registry Severity Index Criteria (IBMTR) to evaluate which organs have been affected and the overall severity of the disease. Ryoncil's effectiveness was based primarily on the rate and duration of response to treatment 28 days after initiating Ryoncil. Study participants who had a partial or mixed response to treatment—meaning that there was improved condition in one organ with either no change (partial) or worsening condition (mixed) in another organ—received additional infusions once weekly for an additional four weeks. Sixteen study participants (30%) had a complete response to treatment 28 days after receiving Ryoncil, while 22 study participants (41%) had a partial response. Infusion of Ryoncil should be monitored by the treating physician, and the infusion should be discontinued if there is any evidence of a reaction which may include dyspnea (shortness of breath), hypotension (low blood pressure), fever, tachypnea (rapid breathing), cyanosis (blue discoloration of skin, lips or nails) and hypoxia (low oxygen in the blood). The most common adverse reactions in study participants who received Ryoncil were infections, fever, hemorrhage, edema, abdominal pain and hypertension. Complications such as hypersensitivity and acute infusion reactions, transmission of infectious disease or agents and ectopic tissue formation may occur following treatment with Ryoncil. Ryoncil is contraindicated in patients with known hypersensitivity to dimethyl sulfoxide or porcine and bovine proteins. Patients should be premedicated with corticosteroids and antihistamines prior to infusion and monitored for hypersensitivity reactions during treatment with Ryoncil. The application received Orphan Drug, Fast Track and Priority Review designations by the FDA. The FDA granted approval of Ryoncil to Mesoblast, Inc. Media Contact: Carly Pflaum, (240) 672-8872 Consumer Inquiries: Email or 888-INFO-FDA The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation's food supply, cosmetics, dietary supplements, radiation-emitting electronic products, and for regulating tobacco products. SOURCE U.S. Food and Drug Administration WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

ROSWELL, Ga. and DUBLIN and HAMILTON, Bermuda, Dec. 3, 2024 /PRNewswire/ -- Saol Therapeutics, a privately held, clinical-stage pharmaceutical company, today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for approval of SL1009, Sodium Dichloroacetate Oral Solution (DCA) for use with a proprietary genetic test, for the treatment of an orphan pediatric mitochondrial disease, Pyruvate Dehydrogenase Complex Deficiency (PDCD)1. SL1009 has been granted Orphan Drug, Fast Track and Rare Pediatric Disease Designations by the FDA and is thus eligible for Priority Review and a Priority Review Voucher. Continue Reading "This NDA submission is an important step in bringing this therapy to children living with PDCD in the U.S., where no approved treatment is currently available," said Dave Penake, Chief Executive Officer of Saol Therapeutics. "We look forward to continuing our ongoing discussions with the FDA as we move closer to addressing this significant unmet need of children suffering from PDCD." Saol Therapeutics today announced the submission of a NDA to the FDA for approval of SL1009, Sodium Dichloroacetate Post this The NDA is supported by results from the Phase 3 double-blind placebo controlled cross-over study (SL1009-01) and a survival study (SL1009-02). The totality of evidence submitted in the NDA includes mechanistic characterization, nonclinical and clinical safety and efficacy evidence to support the clinical benefit of DCA in PDCD patients. The primary endpoint of SL1009-01 was a daily Observer Reported Outcomes survey tool to measure changes in motor domains (ObsROmotor) as well as the safety and tolerability of SL1009 compared to placebo. A key secondary endpoint measured the reduction in plasma lactate of SL1009 compared to placebo. Patients receiving DCA were dose-stratified post-randomization utilizing a proprietary genetic test. The test identifies each patient's GSTZ1 genotype to provide individualized dosing intended to reduce adverse events, such as peripheral neuropathy. A second study, SL1009-02, was a survival analysis that compared outcomes for DCA-treated patients in the Phase III study with external, untreated natural history cohort of patients with PDCD. Patients were matched on age and sex. Peter Stacpoole, MD, initial study sponsor of SL1009-01 said "The NDA submission is a tremendous milestone and the culmination of years of effort. I am grateful for the clinicians and families who participated in the trial." Detailed data is expected to be presented at an upcoming medical conference. DCA is not currently approved for any indication in the United States. About SL1009 (DCA) and GSTZ1/MAAI Proprietary Genetic Test SL1009, if approved by the FDA after a review of safety and efficacy, has the potential to be the 1st approved medication for the mitochondrial disease PDCD and would be available as an oral solution. Gene mutations in the mitochondrial Pyruvate Dehydrogenase Complex (PDC) lead to congenital PDCD. PDC is inhibited by pyruvate dehydrogenase kinases (PDK), that may be over-expressed in PDCD. DCA inhibits PDK to stimulate residual PDC activity and increase energy (ATP) production by mitochondria. Dosing is based on a proprietary genetic test that dichotomizes subjects into "fast" and "slow" drug metabolizers, providing individualized dosing. About Pyruvate Dehydrogenase Complex Deficiency (PDCD) PDCD is a mitochondrial disorder of carbohydrate oxidation that mostly affects the nervous system and skeletal muscle and leads to decreased ATP production and energy failure. It is estimated that 300-500 patients are treated in expert centers in the US, and the overall prevalence is thought to be as high as 2,000. PDCD is the most common cause of congenital lactic acidosis, a life-threatening condition that may occur as early as the neonatal period. Patients suffering from PDCD may also exhibit extreme tiredness (lethargy), poor feeding, rapid breathing (tachypnea), and other signs of neurological and neuromuscular dysfunction such as developmental delay, low muscle tone (hypotonia), abnormal eye movements and seizures. Signs and symptoms usually begin soon after birth but may appear later in childhood2. There are currently no FDA-approved therapies for PDCD. About Saol Therapeutics Saol Therapeutics (pronounced "Sail") is a privately held, clinical-stage, pharmaceutical company with operations in Roswell, GA, Dublin, Ireland and Hamilton, Bermuda. Saol is focused on development activity in CNS disorders such as spasticity and pain management, and orphan diseases. Saol is committed to providing and advancing therapeutic options for patients and the physicians treating these populations. For more information, visit . References , Accessed October 2024 Ganetzky R, McCormick EM, Falk MJ. Primary Pyruvate Dehydrogenase Complex Deficiency Overview. 2021 Jun 17. In: Adam MP, Feldman J, Mirzaa GM, Pagon RA, Wallace SE, Bean LJH, Gripp KW, Amemiya A, editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993–2023. PMID: 34138529. Saol Therapeutics Contact Senior Vice President, Strategy Brian Nappi [email protected] SOURCE Saol Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED