11132020

FDA approves prescription-only Apple Watch app for PTSD nightmares

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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11122020

Surface Oncology Announces FDA Fast Track Designation Granted by U.S. Food and Drug Administration for SRF388 to Treat Liver Cancer

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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11112020

Immune-Onc Therapeutics Announces Orphan Drug Designation of IO-202 (Anti-LILRB4) for Treatment of AML and Poster Presentation at ASH 2020

 “Trials in Progress” poster presentation at the American Society of Hematology (ASH) Annual Meeting to detail mechanisms of action, preclinical data and trial design 

– IO-202, targeting the immune inhibitory receptor LILRB4 (also known as ILT3), is being evaluated in a Phase I trial for acute myeloid leukemia (AML) and chronic myelomonocytic leukemia (CMML) –

Immune-Onc Therapeutics, Inc. (“Immune-Onc”), a clinical-stage cancer immunotherapy company today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation status for its first-in-class antagonist antibody IO-202 for treatment of acute myeloid leukemia (AML). In addition, the company announced acceptance of its “Trials in Progress” poster presentation for IO-202 at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition. The meeting is taking place virtually December 5-8, 2020.

Orphan Drug Designation qualifies the sponsor for various development incentives of the Orphan Drug Act, including exemption of FDA application fees and tax credits for qualified clinical testing, to advance the evaluation and development of products that demonstrate promise for the diagnosis and treatment of rare diseases or conditions that affect fewer than 200,000 people in the United States. Orphan Drug Designation can also convey seven years of marketing exclusivity for a drug approved to treat an orphan disease in the United States.

“Receiving orphan drug designation for IO-202 in AML is another important milestone for Immune-Onc and underscores the need for effective new treatments for this aggressive and hard-to-treat cancer,” said Charlene Liao, Ph.D., chief executive officer of Immune-Onc. “We are pleased with the progress we are making in advancing IO-202. As outlined in our ASH poster presentation, IO-202 holds promise for AML patients because it demonstrates novel mechanisms of action in overcoming immune suppression. IO-202 is one of several programs in our pipeline that target the LILRB family of immune inhibitory receptors. We are excited to continue our momentum in evaluating IO-202 and Immune-Onc’s preclinical candidates in other cancers, including solid tumors, in the near future.”

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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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11102020

Rafael Pharmaceuticals Receives FDA Fast Track Designation for CPI-613® (devimistat) for the Treatment of Pancreatic Cancer

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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11092020

Novavax COVID-19 Vaccine Granted Fast Track Designation by U.S. FDA

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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11062020

FDA grants orphan drug designation to natural killer cell therapy for multiple myeloma

The FDA granted orphan drug designation to CellProtect, a modified natural killer cell therapy for the treatment of patients with multiple myeloma, according to the agent’s manufacturer.

CellProtect (XNK Therapeutics) is an investigational autologous cell therapy produced through ex vivo expansion and activation of natural killer cells from peripheral blood of patients with cancer.

The technology used to produce the therapy is based on natural killer cell research performed at Karolinska Institute in Stockholm. The manufacturing process takes a little less than 3 weeks, according to the manufacturer.

“Obtaining an [orphan drug designation] by the FDA is a significant milestone for XNK and our goal of taking [this] drug candidate to the next level,” Johan Liwing, CEO of XNK Therapeutics, said in a press release. “This is the starting point for us to expand clinical development into the most important market globally for cancer treatment.”

Researchers tested CellProtect in the phase 1/phase 2 ACP-001 trial, results of which were presented at this year’s European Hematology Association Annual Meeting. The trial evaluated CellProtect as consolidation treatment after high-dose stem cell transplantation among patients with newly diagnosed multiple myeloma.

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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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11052020

LogicBio Therapeutics Receives FDA Fast Track Designation for LB-001 for the Treatment of Methylmalonic Acidemia (MMA)

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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11042020

Salvia BioElectronics receives FDA Breakthrough Device Designation for innovative neurostimulation solution

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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11032020

Food and Drug Administration Accepts BioMarin’s New Drug Application for Vosoritide to Treat Children with Achondroplasia

  • If approved, 1st Therapy in U.S. for the Treatment of Achondroplasia
  • PDUFA Action Date is Aug. 20, 2021

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for vosoritide, an investigational, once daily injection analog of C-type Natriuretic Peptide (CNP) for children with achondroplasia, the most common form of disproportionate short stature in humans. This acceptance by the FDA marks the first marketing application accepted for a treatment for achondroplasia in the United States.

The Prescription Drug User Fee Act (PDUFA) action date is Aug. 20, 2021. The FDA has informed the company that they are not currently planning to hold an advisory committee meeting to discuss the application.

Although the FDA did not identify any filing issues with the NDA, the Agency reiterated a position raised during the Pediatric Advisory Committee (PAC) and Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) held on May 11, 2018 recommending two-year controlled trials in different age groups. BioMarin believes the highly persuasive outcomes from the one-year randomized, double-blind, placebo-controlled Phase 3 trial, coupled with data from the Phase 2 program with up to 5 years of long-term follow-up that has been compared to robust natural history data on growth, offers a rigorous and reliable method to assess whether vosoritide has a durable impact on the rate of endochondral bone growth that ultimately increases final adult height. This information was included in the marketing application.

While not part of the vosoritide marketing application, in Q4, the Company is also expecting to complete enrollment in a Phase 2 randomized, placebo-controlled study of vosoritide in approximately 70 infants and young children with achondroplasia, aged zero to less than 60 months, for a period of 52 weeks. The study will be followed by a subsequent open-label extension trial when all subjects receive active treatment. Children in this study will complete a minimum three-month baseline study to determine their respective baseline growth prior to dosing in the Phase 2 study. Children in this study will have completed a minimum three-month baseline study to determine their respective baseline growth prior to entering the Phase 2 study. The primary objectives of the study are to evaluate safety, tolerability, and the effect of vosoritide on height Z-scores, which is the number of standard deviations in relation to the mean height of age-matched, average stature children. The company also plans to augment the height Z-score data with assessments including proportionality, functionality, quality of life, sleep apnea, and foramen magnum dimension, as well as the advent of major illnesses and surgeries.

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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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11022020

Orphan Drug Designation Granted for CSL Behring’s Investigational Plasma-Derived Hemopexin Therapy for Sickle Cell Disease

Global biotherapeutics leader CSL Behring announced today that its investigational, plasma-derived hemopexin therapy (CSL889) received orphan drug designation from both the European Commission and the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development for the treatment of sickle cell disease (SCD). These designations grant special status to drugs and biological products intended to treat a rare disease, affecting less than 200,000 patients in the US or affecting not more than five in 10,000 people in the European Union.

CSL889 is a form of plasma-derived hemopexin, an important, naturally occurring protein produced in the body whose levels are decreased in patients with SCD. Low levels of hemopexin have been associated with increased symptoms in SCD, particularly acute vaso-occlusive crises (VOC). VOC, the most common manifestation in SCD, are severe, debilitating episodes characterized by severe pain. There is no approved treatment for acute VOC, so episodes can only be managed with supportive measures such as fluids and pain killers.

“Having treated hundreds of adults and children living with sickle cell disease over 30 years, I’m intensely aware of the need for novel and effective therapies, especially to relieve the tremendous pain from VOC,” said Professor Greg Kato, who is leading the clinical development of CSL 889 at CSL Behring. “This newly granted orphan status recognizes the urgency for progressing new treatment options into the clinic.”

CSL Behring has two Phase I SCD programs poised to evolve the treatment paradigm for patients: CSL889 hemopexin therapy for the treatment of VOC and CSL200 lentiviral stem cell gene therapy for long-term disease management. 

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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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