New Drug Application

Apollomics Announces Approval of Vebreltinib in China as a First-in-Class Treatment for Gliomas with MET Fusion Gene

Retrieved on: 
Donnerstag, April 25, 2024
Glioma, Shanda, MET, WHO, Temozolomide, Safety, Risk, NMPA, National Medical Products Administration, Glioblastoma, Isocitrate dehydrogenase, Survival, New Drug Application, Cisplatin, Death, Central nervous system, Partnership, Trial of the century, Etoposide, OS, History, CNS, IDH, Patient, Neoplasm, Met, APLM, NSCLC, Lung cancer, Pharmaceutical industry

FOSTER CITY, Calif., April 25, 2024 (GLOBE NEWSWIRE) -- Apollomics Inc. (Nasdaq: APLM), a clinical-stage biopharmaceutical company developing multiple oncology drug candidates to address difficult-to-treat and treatment-resistant cancers, today announced that its partner in China, Avistone Biotechnology Co. Ltd. (Avistone), received approval from the National Medical Products Administration (NMPA) of China for vebreltinib (APL-101) for the treatment of adult patients with isocitrate dehydrogenase (IDH) mutant astrocytoma (WHO Grade 4) or glioblastoma with a history of low-grade disease who have the PTPRZ1-MET fusion (ZM fusion) gene and have failed previous treatments. This supplemental New Drug Application (sNDA) approval makes vebreltinib the world’s first c-Met inhibitor approved for treatment of Central Nervous System (CNS) tumor with c-Met alteration, and follows the NMPA’s November 2023 approval of vebreltinib for the treatment of patients with Met Exon 14 skipping non-small cell lung cancer (NSCLC).

Key Points: 
  • "The NMPA’s approval of vebreltinib in gliomas is an important, first-in-class approval as it demonstrates vebreltinib’s CNS penetration ability and c-Met inhibitory activity in the tumors there," said Guo-Liang Yu, Ph.D., Chairman and Chief Executive Officer of Apollomics.
  • Surgery, radiation treatment, and chemotherapy are current standard treatment strategies for gliomas with poor prognoses.
  • These preliminary data support cross-region similarity of patient response to treatment with vebreltinib.
  • This collaboration enables both companies to leverage their strengths and maximize the benefit of vebreltinib worldwide.

Junshi Biosciences Announces NDA Acceptance in Hong Kong for Toripalimab

Retrieved on: 
Donnerstag, April 25, 2024
PFS, Shirin David, JAMA, Risk, NDA, Government, Progression-free survival, New Drug Application, American Medical Association, Cisplatin, Death, Immunotherapy, Trial of the century, ASCO, European, Health department, Dor, Patient, Nasopharyngeal carcinoma, Society, NPC, Marketing, Clutch (eggs), Journal, Pharmaceutical industry

Toripalimab plus chemotherapy also reduced the risk of disease progression or death by 48% and the risk of death by 37%, all while demonstrating a manageable safety profile.

Key Points: 
  • Toripalimab plus chemotherapy also reduced the risk of disease progression or death by 48% and the risk of death by 37%, all while demonstrating a manageable safety profile.
  • So far, toripalimab has been approved for marketing in the Chinese mainland and the US.
  • Several NDAs are currently under regulatory review in the European Union, UK, Australia, Singapore, Malaysia and Hong Kong Special Administrative Region (“SAR”).
  • Furthermore, NDAs for toripalimab have also been submitted in India, South Africa, Chile and Jordan.

XOMA Earns $9 Million Milestone as FDA Grants Accelerated Approval to Day One’s OJEMDATM (tovorafenib) for Relapsed or Refractory BRAF-altered Pediatric Low-Grade Glioma (pLGG)

Retrieved on: 
Donnerstag, April 25, 2024
FDA, Glioma, NDA, Food, Economics, New Drug Application, BRAF, Patient, Therapy, LGG, BRAF V600, Pharmaceutical industry

EMERYVILLE, Calif., April 25, 2024 (GLOBE NEWSWIRE) -- XOMA Corporation (NASDAQ: XOMA), the biotech royalty aggregator, announced today it has earned a $9 million milestone related to the U.S. Food and Drug Administration’s (FDA) approval of Day One Biopharmaceuticals’ New Drug Application (NDA) for OJEMDA™ (tovorafenib) for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation. 

Key Points: 
  • First and only FDA-approved type II RAF inhibitor for patients with relapsed or refractory pLGG harboring a BRAF fusion or rearrangement, or BRAF V600 mutation
    EMERYVILLE, Calif., April 25, 2024 (GLOBE NEWSWIRE) -- XOMA Corporation (NASDAQ: XOMA), the biotech royalty aggregator, announced today it has earned a $9 million milestone related to the U.S. Food and Drug Administration’s (FDA) approval of Day One Biopharmaceuticals’ New Drug Application (NDA) for OJEMDA™ (tovorafenib) for the treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation.
  • “This is an important milestone for XOMA and our royalty portfolio, but more importantly, it is a watershed event for children living with low-grade gliomas with BRAF alterations that have relapsed or progressed,” stated Owen Hughes, Chief Executive Officer of XOMA.
  • In March 2021, XOMA paid $13.5 million upfront to acquire the $54 million in potential milestones and mid-single digit royalties associated with tovorafenib, plus a share of potential event-based economics, in addition to the economics associated with vosaroxin, from Viracta Therapeutics.

China Medical System: New Drug Application of Desidustat Tablets Accepted in China

Retrieved on: 
Dienstag, April 23, 2024
CMS, Tablet, Desidustat, NMPA, NDA, National Medical Products Administration, Kidney failure, New Drug Application, Erythropoiesis, Anemia, Chronic kidney disease, CKD, Week, Patient, Dialysis, Prevalence, Marketing, Diagnosis, Advertising, EPO, Medical device

Desidustat Tablets are novel, oral HIF-PHI for treating anaemia in non-dialysis adult chronic kidney disease patients.

Key Points: 
  • Desidustat Tablets are novel, oral HIF-PHI for treating anaemia in non-dialysis adult chronic kidney disease patients.
  • The primary endpoint has indicated that Desidustat is more effective than placebo in increasing Hb level.
  • China Medical System Holdings Limited (“CMS” or the “Group”) is pleased to announce that on 22 April 2024, the New Drug Application (NDA) of Desidustat Tablets  (“Desidustat Tablets” or the “Product”) has been accepted by the National Medical Products Administration of China (NMPA).
  • Any treatment-related decisions made by healthcare professionals should be based on the patient’s specific circumstances and in accordance with the drug package insert.

Oculis Strengthens its Executive Leadership Team with the Appointment of Snehal Shah, Pharm.D. as President of Research & Development

Retrieved on: 
Mittwoch, April 10, 2024
FDA, NDA, Food, Pharmacovigilance, OCS, Kyowa Kirin, New Drug Application, Geographic atrophy, Research, Development, Disease, Research and development, Patient, Eye, Paratriathlon, Pharming, Drug development, Ophthalmology

Dr. Shah joins Oculis from Iveric Bio where he served as Chief Regulatory & Product Strategy Officer and led the successful approval of Izervay® for Geographic Atrophy in the U.S.

Key Points: 
  • Dr. Shah joins Oculis from Iveric Bio where he served as Chief Regulatory & Product Strategy Officer and led the successful approval of Izervay® for Geographic Atrophy in the U.S.
  • He has a proven track record of spearheading innovative programs in various therapeutic areas including in ophthalmology for both small molecules and biologics through product development lifecycle phases from conception through commercialization.
  • Riad Sherif, M.D., Chief Executive Officer of Oculis: “We are thrilled to welcome Snehal to Oculis as we continue to expand our U.S. footprint.
  • Snehal’s leadership and development expertise, especially in ophthalmology, will be pivotal to advancing our innovative and differentiated pipeline.

Avistone Announces the Approval of Vebreltinib as the First MET-TKI Treatment for a Rare Brain Glioma Subtype in China

Retrieved on: 
Donnerstag, April 25, 2024
Oncology, Health, Neurology, Clinical Trials, Pharmaceutical, Biotechnology, Glioma, Shanda, MET, Temozolomide, Safety, Radiation, NMPA, Glioblastoma, Isocitrate dehydrogenase, Prognosis, Survival, New Drug Application, Cisplatin, Death, Central nervous system, Lung cancer, Etoposide, OS, History, Risk, CNS, Therapy, IDH, Patient, Neoplasm, NSCLC, Pharmaceutical industry

This supplemental New Drug Application (sNDA) approval makes vebreltinib the world’s first c-Met inhibitor approved for treatment of Central Nervous System (CNS) tumor with c-Met alteration.

Key Points: 
  • This supplemental New Drug Application (sNDA) approval makes vebreltinib the world’s first c-Met inhibitor approved for treatment of Central Nervous System (CNS) tumor with c-Met alteration.
  • In November 2023, vebreltinib received conditional approval for the treatment of metastatic patients with MET Exon14 Skipping non-small cell lung cancer (NSCLC) from the NMPA.
  • Glioma is a refractory primary malignant intracranial tumor, accounting for approximately 46% of intracranial tumors [1].
  • The approval of vebreltinib for the ZM fusion-positive glioma indications is based on the positive results of the FUGEN study (NCT06105619).

Vertex Announces Advancements of Suzetrigine (VX-548) in Acute and Neuropathic Pain

Retrieved on: 
Donnerstag, April 18, 2024
Health, FDA, Neurology, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, VRTX, Pain, Pregabalin, Safety, NDA, Food, New Drug Application, DPN, Sciatica, Fast track (FDA), Week, Patient, Medicine, Neuropathic pain, LSR, Vertex, Vertex Pharmaceuticals, Pharmaceutical industry

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced important advancements across its suzetrigine pain program, which has the potential to be the first new class of medicine for acute and neuropathic pain in more than two decades.

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced important advancements across its suzetrigine pain program, which has the potential to be the first new class of medicine for acute and neuropathic pain in more than two decades.
  • Following the positive Phase 3 results in acute pain announced in January 2024, the Food and Drug Administration (FDA) has granted a rolling New Drug Application (NDA) submission for suzetrigine in moderate-to-severe acute pain.
  • Suzetrigine was previously granted FDA Fast Track and Breakthrough Therapy designations in moderate-to-severe acute pain.
  • The company intends to advance its next generation NaV1.8 pain signal inhibitor VX-993 oral formulation into Phase 2 acute pain and peripheral neuropathic pain studies later this year.

Lumicell’s Cutting-Edge Imaging Platform Receives Historic FDA Approval to Illuminate Residual Breast Cancer

Retrieved on: 
Donnerstag, April 18, 2024
Medical Devices, Surgery, FDA, Clinical Trials, Health Technology, Other Health, Biotechnology, Pharmaceutical, Health, Oncology, Urine, Society, Cancer, Hospital, Safety, Woman, NDA, New Drug Application, Surgeon, Breast, MD, Doctor of Philosophy, Patient, DVS, Anaphylaxis, Hypersensitivity, NEJM, Harvard Medical School, Multimedia, MD Anderson Cancer Center, Surgical oncology, Neoplasm, Massachusetts General Hospital, PMA, Breast cancer, Medical imaging

The most common side effects with LUMISIGHT are hypersensitivity and an abnormal color in urine.

Key Points: 
  • The most common side effects with LUMISIGHT are hypersensitivity and an abnormal color in urine.
  • Results of the Investigation of Novel Surgical Imaging for Tumor Excision (INSITE) pivotal trial, used to support the efficacy of the system, were published in NEJM Evidence .
  • “We want to thank our clinical investigators and the hundreds of women who participated in our breast program for LUMISIGHT and Lumicell DVS,” said Jorge Ferrer, Chief Scientific Officer, Lumicell.
  • “Due to your important contributions, LUMISIGHT and Lumicell DVS are now approved and will be available in the United States shortly.”
    Please visit www.LumiSystem.com to learn more about LUMISIGHT and Lumicell DVS.

Marinus Pharmaceuticals Provides Update on the Phase 3 RAISE Trial and Reports Preliminary First Quarter 2024 Financial Results

Retrieved on: 
Montag, April 15, 2024
Oncology, Health, FDA, Clinical Trials, Research, Science, Pharmaceutical, Epilepsy, Administration, Marinus, Drug discovery, Investment, Safety, Food, Human services, New Drug Application, Development, Data monitoring committee, Therapy, Ganaxolone, Patient, Tuberous sclerosis, DMC, Medication, Pharmaceutical industry

Marinus has decided to complete enrollment in the RAISE trial at approximately 100 patients with topline results expected in the summer of 2024.

Key Points: 
  • Marinus has decided to complete enrollment in the RAISE trial at approximately 100 patients with topline results expected in the summer of 2024.
  • Those results will be used to determine whether to continue development of IV ganaxolone.
  • Marinus expects to complete enrollment in the Phase 3 TrustTSC trial of ZTALMY® (ganaxolone) oral suspension CV with approximately 130 patients in mid-May 2024.
  • Marinus estimates preliminary unaudited cash, cash equivalents, and short-term investments of $113.3 million as of March 31, 2024.

GenFleet Therapeutics Announces FDA's Clinical Trial Approval for GFH925 (KRAS G12C Inhibitor) Monotherapy in Phase III Registrational Study Treating Metastatic Colorectal Cancer

Retrieved on: 
Freitag, April 19, 2024
FDA, Total, Colorectal cancer, Mutation, Immunology, Lung cancer, Priority review, NMPA, National Medical Products Administration, Cetuximab, Progression-free survival, New Drug Application, Irinotecan, Oxaliplatin, Recurrence, RAS, CRC, ESMO, Medicine, ASCO, Canadian International Council, Disease, Incidence, Patient, Liver, Peritoneum, Food and Drug Administration, BTD, Lung, Death, Biology, Clinical trial, EGFR, Therapy, Cancer, NSCLC, Breakthrough therapy, Prevalence, Pharmaceutical industry

"We appreciate the FDA's recognition of GFH925' efficacy and safety profile, as this is our first global phase III monotherapy study in treating advanced stage CRC.

Key Points: 
  • "We appreciate the FDA's recognition of GFH925' efficacy and safety profile, as this is our first global phase III monotherapy study in treating advanced stage CRC.
  • We believe the approval of our proposed trial design highlights the potential of GFH925 monotherapy in this indication, thus bringing more therapeutic options and clinical benefits to the patients.
  • Targeting KRAS G12C Mutation in Colorectal Cancer, A Review: New Arrows in the Quiver, International Journal of Molecular Science, Mar.
  • Disease Burden of Total and Early-Onset Colorectal Cancer in China from 1990 to 2019 and Predictions of Cancer Incidence and Mortality, Clinical Epidemiology, Feb. 2023
    4.