New Drug Application

GH Research Reports First Quarter 2024 Financial Results and Provides Business Updates

Retrieved on: 
Friday, May 3, 2024
FDA, Food, BDII, GHRS, Research, Psychiatry, New Drug Application, Insurance, TRD, Psychotherapy, IND, Patient, DSM-IV codes, Clinical trial, PD, G&A, Rat, Pharmaceutical industry

DUBLIN, Ireland, May 03, 2024 (GLOBE NEWSWIRE) -- GH Research PLC (Nasdaq: GHRS), a clinical-stage biopharmaceutical company dedicated to transforming the treatment of psychiatric and neurological disorders, today reported financial results for the first quarter ended March 31, 2024, and provided updates on its business.

Key Points: 
  • DUBLIN, Ireland, May 03, 2024 (GLOBE NEWSWIRE) -- GH Research PLC (Nasdaq: GHRS), a clinical-stage biopharmaceutical company dedicated to transforming the treatment of psychiatric and neurological disorders, today reported financial results for the first quarter ended March 31, 2024, and provided updates on its business.
  • We continue to expect GH001-PPD-203 completion and availability of top-line data in the third quarter of 2024.
  • R&D expenses were $8.7 million for the quarter ended March 31, 2024, compared to $7.3 million for same quarter in 2023.
  • G&A expenses were $2.9 million for the quarter ended March 31, 2024, compared to $3.1 million for the same quarter in 2023.

Lipocine Announces Completion of Dosing in Pivotal Study of Postpartum Depression Candidate, LPCN 1154

Retrieved on: 
Wednesday, May 1, 2024
FDA, Pregnancy, Woman, PPD, NDA, Food, CDC, New Drug Application, DSM-IV codes, Patient, Physician, Diagnosis, Pharmaceutical industry

SALT LAKE CITY, May 1, 2024 /PRNewswire/ -- Lipocine Inc. (NASDAQ: LPCN), a biopharmaceutical company, today announced that dosing of subjects has been completed in the pivotal pharmacokinetic (PK) study designed to support a New Drug Application (NDA) for LPCN 1154. LPCN 1154, oral brexanolone, is being developed as a treatment of postpartum depression (PPD).  Lipocine anticipates topline results late in the second quarter of 2024, with the goal of NDA submission by the end of the fourth quarter of 2024.

Key Points: 
  • SALT LAKE CITY, May 1, 2024 /PRNewswire/ -- Lipocine Inc. (NASDAQ: LPCN), a biopharmaceutical company, today announced that dosing of subjects has been completed in the pivotal pharmacokinetic (PK) study designed to support a New Drug Application (NDA) for LPCN 1154.
  • LPCN 1154, oral brexanolone, is being developed as a treatment of postpartum depression (PPD).
  • "We are pleased to complete dosing of participants in this NDA enabling study of LPCN 1154," said Dr. Mahesh Patel, President and CEO of Lipocine.
  • Oral LPCN 1154, comprising a bioidentical neuroactive steroid with 48-hour outpatient dosing, is being developed to provide rapid relief with robust efficacy.

Ascendis Pharma Reports First Quarter 2024 Financial Results

Retrieved on: 
Thursday, May 2, 2024
FDA, Endocrine gland, Hypoparathyroidism, Element, GHD, Achondroplasia, Commercial, New Drug Application, Biologics license application, Development, Prescription, ASCO, Turner syndrome, PDUFA, Patient, Society, Melanoma, Vision, Inborn errors of metabolism, Pharmaceutical industry

If approved, U.S. launch planned in Q3

Key Points: 
  • If approved, U.S. launch planned in Q3
    — SKYTROFA® Q1 revenue more than doubled year-over-year to €65 million; Q1 operating expenses fell by 20% year-over-year to €137 million
    COPENHAGEN, Denmark, May 02, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced financial results for the first quarter ended March 31, 2024, and provided a business update.
  • Plan to submit a supplemental Biologics License Application to FDA for adult growth hormone deficiency (GHD), in the third quarter of 2024.
  • Topline results from Phase 2 trial in Turner syndrome expected in the fourth quarter of 2024.
  • In the U.S., Prescription Drug User Fee Act (PDUFA) date of May 14, 2024; if approved, U.S. commercial launch planned in the third quarter of 2024.

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

Retrieved on: 
Thursday, 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: 
Thursday, 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: 
Thursday, 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: 
Tuesday, 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: 
Wednesday, 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: 
Thursday, 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: 
Thursday, 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.