Industry Updates
Stay ahead with curated daily updates from the pharmaceutical and healthcare sectors. Explore key FDA designations, pivotal drug development milestones, and actionable insights from clinical trials—delivered with clarity and strategic relevance to empower your next move.
- 30 Oct 2025
FDA Grants Orphan Drug Designation to Dewpoint Therapeutics’ DPTX-3186 for Gastric Cancer
The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Dewpoint Therapeutics’ investigational candidate DPTX-3186 for the treatment of gastric cancer, a rare and aggressive malignancy with limited therapeutic options. DPTX-3186 is designed to modulate biomolecular condensates—novel cellular structures implicated in disease biology—offering a new approach to targeting cancer pathways. This designation provides regulatory incentives and highlights the potential of condensate biology to unlock innovative treatments for underserved oncology indications.
Thelansis’s “Gastric Cancer – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034″ covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Netherton Syndrome treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).
PDS Biotech Pursues Expedited FDA Approval Pathway for PDS0101 Immunotherapy in HPV16-Positive Head and Neck Cancer
PDS Biotech has announced its intent to pursue expedited FDA approval for PDS0101, an investigational immunotherapy targeting HPV16-positive head and neck cancer. PDS0101 is designed to activate tumor-specific CD8+ T cells against malignancies driven by the HPV16 oncogene. The company’s decision aligns with growing regulatory interest in therapeutic vaccines and underscores the urgent need for effective immunotherapies in HPV-associated cancers.
Thelansis’s “Head and Neck Cancer (HNC) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034″ covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Netherton Syndrome treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).
- 29 Oct 2025
GSK and Empirico Sign Licensing Agreement for Clinical-Stage Oligonucleotide Therapy Targeting Respiratory Diseases
GSK and Empirico entered into a strategic licensing agreement to license and commercialize a first-in-class, clinical-stage oligonucleotide therapy for targets in respiratory disease, including chronic obstructive pulmonary disease (COPD). The candidate, discovered through Empirico’s precision genomics platform, offers a novel approach to regulating gene expression in conditions with high unmet medical need. This partnership deepens GSK’s genomic medicine pipeline and reinforces its commitment to advancing transformative therapies for respiratory diseases—such as COPD—through evidence-driven drug discovery.
Thelansis’s “Chronic Obstructive Pulmonary Disease (COPD) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034″ covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Netherton Syndrome treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).
Neurenatis Secures EMA Orphan Drug Designation for NEU-001 in Hirschsprung Disease
The European Medicines Agency (EMA) has granted Orphan Drug Designation to Neurenatis’ investigational therapy NEU-001 for the treatment of Hirschsprung disease, a rare congenital condition affecting the enteric nervous system. NEU-001 is intended to cure the underlying deficiency of enteric neurons within the colon causing severe gastrointestinal impairment in affected infants and children. The designation offers regulatory incentives and underscores the critical need for personalized treatments for pediatric orphan diseases with limited therapy choices.
Thelansis’s “Hirschsprung Disease (HD) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034″ covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Netherton Syndrome treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).
- 28 Oct 2025
China’s NMPA Grants Breakthrough Therapy Designation to Gotistobart (BNT316/ONC-392) for Advanced sqNSCLC
China’s National Medical Products Administration (NMPA) has granted Breakthrough Therapy Designation to Gotistobart (BNT316/ONC-392), an anti-CTLA-4 antibody developed by BioNTech and OncoC4, for the treatment of advanced or metastatic squamous non-small cell lung cancer (sqNSCLC) in patients who have progressed following prior anti-PD-(L)1 therapy. The designation is based on encouraging clinical evidence suggesting that Gotistobart may offer a novel immunotherapeutic option for individuals with limited alternatives.
Thelansis’s “Squamous Non-Small Cell Lung Cancer (sqNSCLC) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034″ covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Netherton Syndrome treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).
BridgeBio’s BBP-418 Shows Efficacy in Phase 3 FORTIFY Study for Limb-Girdle Muscular Dystrophy Type 2I/R9
BridgeBio Pharma has reported positive topline results from its Phase 3 FORTIFY trial evaluating BBP-418, a small molecule therapy for patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). The trial met its primary endpoint, demonstrating statistically significant improvement in functional outcomes compared to placebo. BBP-418 is designed to enhance ribitol metabolism and restore glycosylation of alpha-dystroglycan, a critical protein for muscle integrity. These findings position BBP-418 as a precision therapy for a genetically defined subset of LGMD—a progressive neuromuscular disorder with no approved treatments. BridgeBio plans to advance toward regulatory submission based on these data.
Thelansis’s “Limb-Girdle Muscular Dystrophy Type 2i (LGMD2i) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034″ covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Netherton Syndrome treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).
Alphamab Oncology’s JSKN003 Earns FDA Fast Track Designation for Advanced PROC
Alphamab Oncology’s investigational HER2-targeting antibody-drug conjugate, JSKN003, has received FDA Fast Track designation for the treatment of advanced or metastatic platinum-resistant ovarian cancer, including primary peritoneal and fallopian tube cancers. This designation supports expedited development based on promising early clinical data and highlights the urgent need for novel therapies in hard-to-treat gynecologic malignancies.
Thelansis’s “Platinum-Resistant Advanced Ovarian Cancer – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034″ covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Netherton Syndrome treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).
- 27 Oct 2025
NeurAxis Secures First-Ever FDA Clearance for Treating Pain and Nausea in Functional Dyspepsia
NeurAxis has received the U.S. FDA’s first-ever clearance for a therapy specifically designed to treat pain associated with Functional dyspepsia (FD) and its related nausea symptoms in adults. This milestone represents a significant advancement in the management of FD—a chronic gastrointestinal disorder marked by upper abdominal discomfort and impaired gastric motility. The FDA-approved treatment leverages non-invasive neuromodulation of the gut-brain axis to provide a new, drug-free solution for patients with limited therapeutic alternatives. The action is a reflection of increasing awareness of the benefits of innovative alternatives in functional GI disorders where standard pharmacologic alternatives are either inadequate or absent.
Thelansis’s “Functional Dyspepsia – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034″ covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Netherton Syndrome treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).
- 24 Oct 2025
FDA Supports HDP-101 Development in Relapsed or Refractory Multiple Myeloma with Fast Track Status
Heidelberg Pharma’s HDP-101 has received FDA Fast Track designation for the treatment of relapsed or refractory multiple myeloma. HDP-101 is an antibody-drug conjugate (ADC) that targets BCMA, a protein commonly expressed on malignant plasma cells. The therapy leverages the cytotoxic payload amanitin to selectively eliminate cancer cells while sparing healthy tissue. The Fast Track designation reflects the urgent need for new treatment options in heavily pretreated multiple myeloma patients and supports expedited development and regulatory review of this novel therapeutic approach.
Thelansis’s “Relapsed or Refractory Multiple Myeloma – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034″ covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Netherton Syndrome treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).
FDA Grants Rolling NDA Review for Egetis’ Emcitate (Tiratricol) in MCT8 Deficiency
Egetis Therapeutics has received a rolling New Drug Application (NDA) review from the U.S. FDA for Emcitate (tiratricol), a thyroid hormone analogue being developed for the treatment of MCT8 deficiency. This rare genetic disorder impairs thyroid hormone transport into the brain, leading to severe neurodevelopmental delays and progressive disability. Rolling review permits Egetis to submit sections of its NDA on an as-completed basis, which could reduce the regulatory timeline. Based on currently available clinical data, this decision reflects the FDA’s recognition of Emcitate’s potential to address a serious unmet medical need in a vulnerable patient population.
Thelansis’s “Allan-Herndon-Dudley Syndrome (AHDS) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034″ covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Netherton Syndrome treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).
- 23 Oct 2025
ADARx Pharmaceuticals Initiates Phase 3 Trial of ADX-324 for Hereditary Angioedema (HAE); Gains Orphan Drug Status
Adarx Pharmaceuticals has initiated its Phase 3 STOP-HAE clinical trial, dosing the first patient with ADX-324—a long-acting siRNA therapy developed for the prophylactic treatment of hereditary angioedema (HAE). In parallel, the U.S. FDA has granted Orphan Drug Designation to ADX-324, recognizing its potential to address this rare and serious condition with limited long-term treatment options. Designed to provide sustained protection against HAE attacks, the therapy leverages targeted RNA interference to suppress disease-driving pathways.
Thelansis’s “Hereditary Angioedema (HAE) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034″ covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Netherton Syndrome treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).
Electra’s ELA026 Receives FDA Breakthrough and EMA PRIME Designations for Secondary HLH
Electra Therapeutics has received both U.S. FDA Breakthrough Therapy and European Medicines Agency (EMA) Priority Medicines (PRIME) designations for ELA026, its investigational monoclonal antibody targeting secondary hemophagocytic lymphohistiocytosis (sHLH). These twin designations highlight the therapy’s potential to address a rare and potentially fatal immune disorder characterized by excessive inflammation and dysregulated immune cell activation. The regulatory support is intended to expedite clinical development and review, aligning with Electra’s goal of delivering a targeted treatment for patients with secondary HLH who currently face limited therapeutic options.
Thelansis’s “Hemophagocytic Lymphohistiocytosis (HLH) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034″ covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Netherton Syndrome treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).
- 22 Oct 2025
Quoin Pharma's QRX003 Gets FDA Orphan Drug Status for Ultra-Rare Netherton Syndrome
Quoin Pharmaceuticals Ltd, a late clinical-stage specialty pharmaceutical company focused on addressing rare and orphan diseases, has announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to its lead product candidate, QRX003, for the treatment of Netherton Syndrome (NS). The ODD, which follows EMA’s May 2025 grant, provides development incentives and fast-tracks QRX003’s path to potentially becoming the first therapy approved for the life-altering Netherton Syndrome.
Thelansis’s “Netherton Syndrome (NS) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034″ covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Netherton Syndrome treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).
FDA Grants Fast Track to Immusoft’s ISP-001, a First-in-Class B-Cell Therapy for MPS I
Immusoft has announced that the U.S. FDA has granted Fast Track designation to ISP-001, its first-in-class engineered B-cell therapy for mucopolysaccharidosis type I (MPS I). This rare lysosomal disorder currently has limited treatment options, and ISP-001 introduces a novel approach by programming patient-derived B cells to continuously produce therapeutic proteins. The designation enables accelerated development and regulatory review, underscoring the therapy’s potential to address serious unmet medical needs.
Thelansis’s “Hurler Syndrome (Mucopolysaccharidosis Type I) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2024 To 2034″ covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Netherton Syndrome treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).
Need customize support?
Thelansis also caters to your specific needs by providing customized reports that satisfy your organizational requirements. Get on-demand customization of the report’s scope to exactly meet your needs.