PRNewswire | July 18, 2023
Karyopharm Therapeutics Inc. a commercial-stage pharmaceutical company pioneering novel cancer therapies, today announced that the United States Food and Drug Administration (FDA) has granted Fast Track Designation to the development program of selinexor for the treatment of patients with myelofibrosis, including primary myelofibrosis, post-essential thrombocythemia myelofibrosis, and post-polycythemia vera myelofibrosis.
"Fast Track Designation for selinexor highlights its potential to address the unmet medical need in myelofibrosis, an important acknowledgement as we continue our pivotal Phase 3 study," said Reshma Rangwala, MD, PhD, Chief Medical Officer of Karyopharm. "Selinexor's unique mechanism of action, XPO1 inhibition, is a novel and potentially fundamental mechanism in myelofibrosis. We have been highly encouraged by the efficacy and safety data observed to date [in our Phase 1 study] with selinexor in combination with ruxolitinib in patients with treatment-naïve myelofibrosis and believe selinexor has the potential to shift the treatment paradigm. We look forward to continued interaction with the FDA as we advance the development of this promising treatment for patients in need."
In June 2023, Karyopharm initiated a pivotal Phase 3 clinical trial to assess the efficacy and safety of once-weekly selinexor 60 mg in combination with ruxolitinib in JAKi-naïve patients with myelofibrosis. Updated data from the Phase 1 study were presented at the American Association for Cancer Research Annual Meeting 2023, American Society of Clinical Oncology 2023 and European Hematology Association 2023, which showed rapid, deep and sustained spleen responses and robust symptom improvement in patients treated with selinexor 60 mg in combination with ruxolitinib as of the April 10, 2023 cut-off date. Top-line data from the Phase 3 study is expected in 2025. The Company plans to expand its clinical development program in myelofibrosis by investigating selinexor in other JAKi-naïve settings, such as novel combinations, to benefit the greatest number of patients.
Fast Track Designation is intended to facilitate development and expedite review of drugs to treat serious and life-threatening conditions so that an approved product can reach the market expeditiously. Features of Fast Track Designation include frequent interactions with the FDA review team, and if relevant criteria are met, eligibility for Priority Review and Rolling Review.
About Karyopharm Therapeutics
Karyopharm Therapeutics Inc. is a commercial-stage pharmaceutical company pioneering novel cancer therapies. Since its founding, Karyopharm has been an industry leader in oral Selective Inhibitor of Nuclear Export (SINE) compound technology, which was developed to address a fundamental mechanism of oncogenesis: nuclear export dysregulation. Karyopharm's lead SINE compound and first-in-class, oral exportin 1 (XPO1) inhibitor, XPOVIO® (selinexor), is approved in the U.S. and marketed by the Company in three oncology indications and has received regulatory approvals in various indications in a growing number of ex-U.S. territories and countries, including Europe and the United Kingdom (as NEXPOVIO®) and China. Karyopharm has a focused pipeline targeting multiple high unmet need cancer indications, including in multiple myeloma, endometrial cancer, myelodysplastic neoplasms and myelofibrosis.
Businesswire | July 26, 2023
Nexus Pharmaceuticals announced it has received U.S. Food and Drug Administration (FDA) approval for Sterile Water for Injection, USP in 10mL and 20mL Single-Dose Vials.
According to the FDA and the FD&C Act, the product is considered bioequivalent and therapeutically equivalent to the reference listed drug (RLD), Sterile Water for Injection USP, of Hospira, Inc. Furthermore, the FDA has granted CGT (competitive generic therapy) designation for the 20mL presentation of sterile water for injection.
“We are proud to be a force for change in the pharmaceutical industry, and we are grateful to the FDA for recognizing the importance of this critical-need product,” said Aman Ahmed, Director of Government Relations and International Sales at Nexus. “The approval of this product is an important step in continuing our efforts to alleviate drug shortages and bring pharmaceutical manufacturing back to the U.S.”
Nexus Pharmaceuticals’ Sterile Water for Injection will be available in cartons of 25 vials.
About Sterile Water for Injection, USP
Sterile Water for Injection, USP is a sterile, nonpyrogenic preparation of water for injection which contains no bacteriostat, antimicrobial agent or added buffer and is supplied only in single-dose containers to dilute or dissolve drugs for injection.
This parenteral preparation is indicated only for diluting or dissolving drugs for intravenous, intramuscular or subcutaneous injection, according to instructions of the manufacturer of the drug to be administered.
About Nexus Pharmaceuticals LLC
Nexus Pharmaceuticals LLC., a US-based healthcare company, specializes in innovative processes to make difficult-to-manufacture specialty and generic drugs that are easier to use, less labor intensive, and more streamlined in practice. Nexus ensures that its high-quality FDA-approved drugs fulfill a critical unmet medical need and delivers dependable life-saving treatment options when and where they’re needed most.
Globenewswire | July 20, 2023
Catalyst Pharmaceuticals, Inc. announced the completion of its acquisition from Santhera Pharmaceuticals Holdings of an exclusive license for North America for vamorolone, a potential treatment for patients suffering with Duchenne Muscular Dystrophy. The license is for exclusive commercial rights in the U.S., Canada, and Mexico, as well as the right of first negotiation in Europe and Japan should Santhera pursue partnership opportunities. Additionally, Catalyst will hold North American rights for any future approved indications of vamorolone.
Vamorolone is a promising best-in-class dissociative anti-inflammatory steroid treatment for Duchenne Muscular Dystrophy ("DMD"). In clinical studies, vamorolone demonstrated efficacy with a significant reduction of steroid-associated side effects and benefits for bone health, growth, and behavior, offering the potential to address an important unmet medical need in DMD patients. Vamorolone has received FDA Orphan Drug and Fast Track designations and has been granted a PDUFA action date of October 26, 2023.
"With the addition of vamorolone, we have accomplished another important milestone in the execution of our portfolio expansion strategy," stated Patrick J. McEnany, Chairman and CEO of Catalyst. "The license for vamorolone reflects our strategic focus on opportunities where we can not only apply novel technology to address critical unmet patient needs, but where we can leverage Catalyst's existing integrated capabilities and infrastructure to commercialize the drug efficiently. We believe that vamorolone, if approved, has the potential to deliver significant near and long-term value and to be a very meaningful advancement to the current standard of care paradigm for DMD patients."
Mr. McEnany continued, "Vamorolone fortifies our neuromuscular portfolio with an innovative therapy that, in clinical studies, demonstrated an enhanced safety and tolerability profile as compared to prednisone. We plan to include vamorolone for DMD patients in our Catalyst Pathways® specialty pharmacy program to help ensure that all patients have access to the full patient benefits of the program. Our planned strategy to facilitate access to vamorolone underscores our steadfast commitment to improving the lives of patients suffering from rare neurological conditions."
Duchenne Muscular Dystrophy, or DMD, the most common form of muscular dystrophy, is a rare, fatal neuromuscular disorder characterized by progressive muscular dysfunction, leading to loss of ambulation, respiratory failure, and death. Corticosteroids are the current standard of care for treating DMD; however, this treatment is associated with significant side effect burdens. The U.S. prevalence for DMD is estimated to be between 11,000 and 13,000 patients. Of patients currently being treated for DMD, approximately 75% receive concomitant steroid treatment.
Vamorolone Commercial Operational Plan
Catalyst expects to launch vamorolone early in the first quarter of 2024, assuming regulatory approval on the PDUFA date of October 26, 2023. Catalyst anticipates minimal sales and marketing personnel expansion with fewer than 10 additional team members required, resulting from the exceptional synergy within its existing neuromuscular franchise. Catalyst plans to incorporate vamorolone for DMD into its Catalyst Pathways® specialty pharmacy program to ensure that patients have access to full patient benefits.
Vamorolone is an investigational drug candidate with a mode of action based on binding to the same receptor as glucocorticoids but modifying its downstream activity and as such, is considered a dissociative anti-inflammatory steroid drug [2-5]. This mechanism has shown the potential to 'dissociate' efficacy from steroid safety concerns, and therefore vamorolone could emerge as an alternative to existing corticosteroids, the current standard of care in children, adolescent, and adult patients with DMD. In the pivotal VISION-DMD study, vamorolone met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile . The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, vomiting, and vitamin D deficiency. Adverse events were generally of mild to moderate severity.
Vamorolone has been granted Orphan Drug status for DMD in the U.S. and Europe and has received Fast Track and Rare Pediatric Disease designations by the U.S. FDA and Promising Innovative Medicine (PIM) status from the UK MHRA for DMD. Vamorolone is an investigational medicine and is currently not approved for use by any health authority.
About Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy ("DMD") is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. DMD is characterized by muscle inflammation and damage which are present at birth or shortly thereafter. Inflammation leads to fibrosis of muscle and is clinically manifested by progressive muscle degeneration and weakness. Major milestones in the disease are the loss of ambulation, the loss of self-feeding, the start of assisted ventilation, and the development of cardiomyopathy. DMD reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure. Corticosteroids are the current standard of care for the treatment of DMD.
About Catalyst Pharmaceuticals
With exceptional patient focus, Catalyst is committed to developing and commercializing innovative first-in-class medicines that address rare neurological and epileptic diseases. Catalyst's U.S. commercial product portfolio consists of FIRDAPSE® (amifampridine) Tablets 10 mg, approved for the treatment of Lambert-Eaton myasthenic syndrome ("LEMS") for adults and children ages six to seventeen. In January 2023, Catalyst acquired the U.S. commercial rights of FYCOMPA® (perampanel) CIII, a prescription medicine approved in people with epilepsy aged four and older alone or with other medicines to treat partial-onset seizures with or without secondarily generalized seizures, and with other medicines to treat primary generalized tonic-clonic seizures for people with epilepsy aged 12 and older. Further, Canada's national healthcare regulatory agency, Health Canada, has approved the use of FIRDAPSE for the treatment of adult patients in Canada with LEMS.