Acetaminophen

Unlike a post-review rejection in the form of a complete response letter, an refuse to file letter means the FDA has declined to evaluate Savara’s application as submitted. Despite hiring a commercial chief and sponsoring disease awareness efforts last year, Savara will have to wait a while longer for a potential approval of its inhaled lung disease asset molgramostim.The FDA has slapped Savara with a refuse to file (RTF) letter after the biotech in March applied for approval of molgramostim in the rare disease autoimmune pulmonary alveolar proteinosis (aPAP).Unlike a post-review rejection in the form of a complete response letter, an RTF letter means the FDA has declined to evaluate Savara’s application as submitted.The FDA has determined that Savara’s initial filing was “not sufficiently complete to permit substantive review,” the biotech said in a Monday press release. The regulator has in turn asked Savara to provide additional chemistry, manufacturing and controls (CMC) data, suggesting some sort of production hang-up played a part in the FDA’s decision.The FDA did not flag any safety concerns related to molgramostim, nor did it request or recommend additional efficacy studies, Savara explained. Savara plans to request a meeting with the FDA in the next 30 days.“Based on our understanding of the letter, we are confident we can thoroughly address the agency’s request and expect to resubmit our [biologics license application] in the fourth quarter of 2025,” Savara CEO Matt Pauls said in a statement. The company is already in the process of generating the CMC data requested by the FDA, Pauls added.“We remain highly confident in our program for autoimmune PAP and believe that our clinical data demonstrate that Molbreevi improves pulmonary gas transfer and respiratory health-related quality of life in this rare disease,” the CEO said, referring to molgramostim by its intended trade name.Savara is developing molgramostim to treat aPAP, a rare disease that clogs the air sacs in the lungs, potentially leading to serious complications like lung fibrosis and the need for a lung transplant. As a granulocyte-macrophage colony-stimulating factor, molgramostim is designed to work by clearing surfactant—a mixture of fat and proteins in the lungs—from the alveoli, or air sacs, in the organs.The drug is delivered via an investigational nebulizer specifically designed by Germany’s PARI Pharma to work with molgramostim, Savara said in its release. Savara has been working on molgramostim for years now, facing various struggles along the way, though the company appeared to believe an approval was in reach as it started making commercial preparations last year.In September, Savara hired Orchard Therapeutics veteran Braden Parker as its chief commercial officer. Parker was primarily brought on to assist with molgramostim’s potential launch, which the company had positioned at the time for late 2025 or 2026.And, in October, Savara also sponsored an episode of “The Balancing Act," a morning talk show that airs on Lifetime, highlighting the stories of patients with rare and genetic diseases. The primary focus of the sponsored program was aPAP, molgramostim’s intended indication.Savara’s continued devotion to molgramostim comes after the company underwent a major pipeline shake-up in 2020, during which it scrapped multiple other assets to hone its focus on aPAP. 

TEL AVIV, Israel, May 27, 2025 (GLOBE NEWSWIRE) -- SciSparc Ltd. (Nasdaq: SPRC) ("Company" or "SciSparc"), a specialty clinical-stage pharmaceutical company focusing on the development of therapies to treat disorders and rare diseases of the central nervous system, announced today the publication of a Japanese divisional patent. The patent application introduces a novel pharmaceutical combination of paracetamol and palmitoylethanolamide (PEA) that may enhance pain relief and fever relief with lower doses and fewer side effects compared to paracetamol monotherapy, with potential for safer and more effective treatment for millions worldwide. “This patent represents a significant innovation in pain management, that potentially could offer a safer alternative for patients of all ages for using this very common drug,” said Oz Adler, Chief Executive Officer of SciSparc. “This drug candidate combines paracetamol, a widely used pain reliever and fever reducer, with PEA, a natural compound that boosts the body’s endocannabinoid system. By working synergistically, the combination may reduce the required paracetamol dose, minimizing risks like liver damage while improving outcomes for acute, chronic, and neuropathic pain, as well as fever.” About SciSparc Ltd. (Nasdaq: SPRC): SciSparc Ltd. is a specialty clinical-stage pharmaceutical company led by an experienced team of senior executives and scientists. SciSparc’s focus is on creating and enhancing a portfolio of technologies and assets based on cannabinoid pharmaceuticals. With this focus, the Company is currently engaged in the following drug development programs based on THC and/or non-psychoactive cannabidiol: SCI-110 for the treatment of Tourette Syndrome, for the treatment of Alzheimer's disease and agitation; and SCI-210 for the treatment of autism and status epilepticus. The Company also owns a controlling interest in a subsidiary whose business focuses on the sale of hemp seeds’ oil-based products on the Amazon.com Marketplace. Forward-Looking Statements: This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995 and other Federal securities laws. For example, the Company is using forward looking statements when discussing the potential benefits and advantages of the novel pharmaceutical combination of paracetamol and PEA and that the patent represents a significant innovation in pain management, that potentially could offer a safer alternative for patients of all ages for using this very common drug. Since such statements deal with future events and are based on SciSparc’s current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of SciSparc could differ materially from those described in or implied by the statements in this press release. The forward-looking statements contained or implied in this press release are subject to other risks and uncertainties, including those discussed under the heading "Risk Factors" in SciSparc's Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (the “SEC”) on April 24, 2025, and in subsequent filings with the SEC. Except as otherwise required by law, SciSparc disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date they were made, whether as a result of new information, future events or circumstances or otherwise. Investor Contact: IR@scisparc.com Tel: +972-3-6167055 Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.