Posts tagged: pediatric rare disease voucher

The majority of post-traumatic stress disorder (PTSD) patients suffer from frequent traumatic nightmares that can deeply disrupt sleep patterns and have been linked with significantly higher rates of suicide. The U.S. Food and Drug Administration (FDA) has now approved a platform called NightWare, designed to help improve sleep in PTSD patients suffering from such recurrent nightmares. The system is an app that runs on Apple Watches and will only be available by prescription.

“Disruptive and persistent traumatic nightmares associated with post-traumatic stress are significantly underreported and undertreated, and there are no consistently effective treatment options,” explains retired US Army General and NightWare advisor Peter Chiarelli. “A device such as the NightWare therapeutic platform that performs the necessary reconnaissance of the mind to identify and interrupt post-traumatic stress-associated nightmares represents a transformational step forward in how we can begin to better support our veterans and active service members suffering from this disabling condition.”

The NightWare system is essentially a sophisticated app that works with an Apple Watch and linked smartphone, and initially spends up to 10 nights learning a user’s sleep patterns. Once a unique sleep profile has been created, the system tracks heart rate and body movement data to detect when a user is experiencing a nightmare. If a nightmare is detected the system triggers mild vibrations through the Apple Watch that are designed to gently disrupt the nightmare without waking the user.

The system was tested in a sham-controlled clinical trial that recruited 70 PTSD patients. The control group received exactly the same intervention as the active group. They used the app and wore a connected Apple Watch, except they received no vibratory stimulation.

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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Surface Oncology (Nasdaq: SURF), a clinical-stage immuno-oncology company developing next-generation immunotherapies that target the tumor microenvironment, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to SRF388 for the treatment of patients with hepatocellular carcinoma (HCC), or liver cancer, who have been previously treated with standard therapies, such as vascular endothelial growth factor targeted agents and programmed death-ligand (PD-L1) blockade.

“Liver cancer is the most rapidly increasing type of cancer in both men and women in the U.S., with incidences tripling since 1980.^1 2 There is a significant need to expedite the development of new therapies to treat liver cancer as the five-year survival for patients with unresectable or metastatic liver cancer is less than five percent,”² said Rob Ross, M.D., chief medical officer. “SRF388 targets IL-27, an immuno-suppressive cytokine that has been found to be elevated in patients with liver cancer, as well as kidney cancer, and we believe SRF388 has the potential to be an effective treatment option for these patients, as monotherapy or in combination with anti-PD-1 therapies.”

SRF388 is currently enrolling patients with advanced solid tumors in a Phase 1 monotherapy dose escalation study with planned expansions in liver and kidney cancer to further evaluate SRF388 as a monotherapy and in combination with other cancer therapies.

The FDA’s Fast Track designation is designed to facilitate the development and expedite the review of drugs that are being developed to treat serious conditions and fill an unmet medical need. The purpose of the designation is to bring important new drugs to patients earlier across a wide range of diseases.

SRF388 recently received orphan-drug designation for treatment of hepatocellular carcinoma from the FDA.

About SRF388:

SRF388 is a fully human anti-IL-27 antibody designed to inhibit the activity of this immuno-suppressive cytokine. Surface Oncology has identified particular tumor types, including liver and kidney cancer, where IL-27 appears to play an important role in the immuno-suppressive tumor microenvironment and may contribute to resistance to treatment with checkpoint inhibitors. SRF388 targets the rate-limiting p28 subunit of IL-27, and preclinical studies have shown that treatment with SRF388 blocks the immuno-suppressive biologic effects of IL-27, resulting in immune cell activation in combination with other cancer therapies and potent anti-tumor effects as a monotherapy. Furthermore, Surface Oncology has identified a potential biomarker associated with IL-27 that may be useful in helping identify patients most likely to respond to SRF388.

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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Rafael Pharmaceuticals, Inc. (“Rafael” or the “Company”), a leader in the growing field of cancer metabolism-based therapeutics, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the Company’s lead compound, CPI-613® (devimistat), for the treatment of metastatic pancreatic cancer. 

“Receiving Fast Track designation is a significant milestone in our fight against pancreatic cancer,” said Sanjeev Luther, President and CEO of Rafael. “This designation further stresses the severe unmet need in treatment options for this aggressive and devastating disease. We are truly thankful to the doctors, researchers, the FDA and all of our supporters who have made this possible. And most of all, we are grateful to the patients involved in our trials; the patients are the inspiration and driving force behind all of our efforts.”  

Rafael has continued to reach milestones throughout the year, including achieving its target enrollment of 500 patients for its Phase 3 trial for metastatic pancreatic cancer ahead of schedule. The Company also recently announced that the FDA granted devimistat Orphan Drug Designation for the treatment of soft tissue sarcoma.  

“Pancreatic cancer is notoriously challenging to treat and long overdue for a new approach,” said Philip A. Philip, M.D., Ph.D., FRCP, Professor of Oncology at the Barbara Ann Karmanos Cancer Institute at Wayne State University and a medical advisor to Rafael. “We have remained hopeful throughout our pancreatic cancer trials, and now with Fast Track designation, our optimism is further fueled. We believe with this designation, cancer metabolism is truly being propelled forward, with devimistat at the helm.”

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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Novavax, Inc. (Nasdaq: NVAX), a late-stage biotechnology company developing next-generation vaccines for serious infectious diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for NVX-CoV2373, the Company’s COVID-19 vaccine candidate. Currently in late-phase clinical development, NVXCoV2373 is a stable, prefusion protein made using Novavax’ nanoparticle technology and includes its proprietary MatrixM™ adjuvant.

“The FDA’s decision to grant Fast Track Designation for NVX-CoV2373 reflects the urgent need for a safe and effective vaccine to prevent COVID-19, and we look forward to working closely with the agency to accelerate access to this vaccine,” said Gregory M. Glenn, M.D., President of Research and Development, Novavax. “While the regulatory review of this clinical program will be expedited, Novavax remains committed to a data-driven and scientifically rigorous approach in demonstrating safety and efficacy, which we believe will support confidence in the vaccine in the U.S. and globally.”

Novavax expects to begin its pivotal Phase 3 clinical trial in the United States and Mexico by the end of November. Data from the event-driven trial could support global authorization and approval, including in the U.S. The Company’s ongoing Phase 3 clinical trial in the UK to evaluate the efficacy, safety and immunogenicity of NVX-CoV2373 is expected to be fully enrolled by the end of November. Depending on the overall COVID-19 attack rate, interim data in the UK trial, which is also event-driven, are expected as soon as early first quarter 2021.

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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LogicBio Therapeutics, Inc. (Nasdaq:LOGC) (LogicBio), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, announced today the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its clinical candidate, LB-001 for the treatment methylmalonic acidemia (MMA). According to the FDA, the purpose of Fast Track designation is to get important new drugs to patients earlier by facilitating the development, and expediting the review, of drugs to treat serious conditions and fill an unmet medical need.

Commenting on the announcement, Daniel Gruskin, M.D., Senior Vice President, Head of Clinical Development of LogicBio, said, “We are pleased the FDA has granted Fast Track designation to LB-001 in recognition of the importance of our efforts to bring a durable treatment to the children suffering from MMA. With Fast Track status, we plan to continue to work closely with the FDA to fully utilize the opportunities presented by this designation to make LB-001 available to patients as quickly as possible.”

About Fast Track Designation

The FDA’s Fast Track designation is a process designed to expedite or facilitate the review of product candidates to treat serious conditions and fill an unmet medical need. Fast Track designation allows for early and frequent communication with the FDA throughout the entire drug development and review process. It may also allow for priority or rolling review of a company’s Biologics License Application (BLA).

About LogicBio Therapeutics

LogicBio Therapeutics is dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms. LogicBio’s proprietary genome editing technology platform, GeneRide, enables the site-specific integration of a therapeutic transgene without nucleases or exogenous promoters by harnessing the native process of homologous recombination. LogicBio has received FDA clearance for the first-in-human clinical trial of LB-001, a wholly owned genome editing program leveraging GeneRide for the treatment of methylmalonic acidemia. Patient enrollment is expected to begin in early 2021. In addition, LogicBio has a collaboration with Takeda to research and develop LB-301, an investigational therapy leveraging GeneRide for the treatment of the rare pediatric disease Crigler-Najjar syndrome.

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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Salvia BioElectronics B.V. (“Salvia”), a neurostimulation platform company targeting chronic migraine, announced today that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Device Designation for its implantable neurostimulation system to address chronic migraine.

Salvia is developing an innovative neurostimulation solution for chronic migraine based on a novel device concept with unique benefits to both patients and physicians. Migraine is the first cause of disability in under 50s, affecting one out of seven people, predominantly women^[1]. People with migraine experience episodes of throbbing, pulsating pain, sometimes accompanied by nausea, vomiting, and sensitivity to light, that can last anywhere from a few hours to a few days. More than five percent of patients suffer from chronic migraine, where they experience migraines for an average of 22 days per month^[2]. Migraine does not only impact wellbeing; it has an enormous impact on work, school, family and social lives.

Wim Pollet, Chief Medical Officer of Salvia BioElectronics, noted: “The FDA breakthrough device designation of our neurostimulation system reflects the recognition of the large unmet medical need of patients suffering from refractory chronic migraine, and the potential of Salvia’s bioelectronic foil technology to address this. We look forward to working closely with the FDA to expedite the review process to accelerate the development of our therapy.”

Patients with chronic migraine suffer from 15 or more headache days per month, with an average of 22 days per month, despite best medical treatment¹. Only 1 in 3 of drug-refractory chronic patients are helped with the newest generation of anti-migraine drugs, leaving many patients in medical need.

While neurostimulation has been demonstrated to be effective for these patients, there are no approved devices commercially available. Salvia was founded with the mission to help these patients suffering from chronic migraine by developing thin and conforming bioelectronic foils that uniquely adapt to the anatomy of the head.

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Disclaimer: BioPharma Global is not responsible for, and expressly disclaims all liability for, damages of any kind arising out of use, reference to, or reliance on any information contained within the article. Content available through the site may contain links and information to other websites. Links from BioPharma Global to third-party sites do not constitute an endorsement by BioPharma Global of the mentioned parties.

BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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