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Jaguar Health Submits Orphan Drug Designation Application to FDA for Crofelemer for Cancer Therapy-Related Diarrhea (CTD) in Patients with Breast Cancer with Metastasis Identified by FDA as a Distinct Condition
Accessnewswire· 2025-09-24 13:00
Potential treatment for patients with breast cancer with metastasis to the brain with crofelemer would augment the significant responder analysis results for crofelemer in the prespecified subgroup of patients with breast cancer from the OnTarget study that were reported at the Multinational Association of Supportive Care in Cancer 2025 Annual Meeting and the 2024 San Antonio Breast Cancer Symposium SAN FRANCISCO, CA / ACCESS Newswire / September 24, 2025 / Jaguar Health (NASDAQ:JAGX) today announced that J ...
Pulmovant Receives Orphan Drug Designation in Japan for Mosliciguat for the Treatment of Pulmonary Hypertension Associated with Interstitial Lung Disease (PH-ILD)
Newsfilter· 2025-09-04 11:00
Core Insights - Pulmovant has received orphan drug designation for mosliciguat from Japan's Ministry of Health, Labour and Welfare, highlighting the significant unmet medical need for patients with Pulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD) [1][2] - Mosliciguat is a novel, once-daily, inhaled soluble guanylate cyclase (sGC) activator currently in Phase 2 clinical trials, aiming to provide a targeted treatment option for PH-ILD [1][3] Company Overview - Pulmovant is a clinical-stage biotechnology company focused on developing treatments for pulmonary diseases and is a subsidiary of Roivant [7] - The company’s lead candidate, mosliciguat, is designed to be a first-in-class inhaled treatment for PH-ILD, with a differentiated mechanism of action [7][5] Clinical Development - Mosliciguat is currently being evaluated in the Phase 2 PHocus clinical study, which is a randomized, double-blind, placebo-controlled trial involving approximately 120 adult patients [3][6] - The Phase 1b ATMOS study demonstrated that inhaled mosliciguat was well tolerated and resulted in a mean peak reduction in pulmonary vascular resistance (PVR) of up to 38% [5] Market Context - PH-ILD is a progressive and life-threatening condition affecting up to 200,000 patients in the U.S. and Europe, with limited or no approved treatment options available [4] - The orphan drug designation provides Pulmovant with regulatory benefits, including priority consultation and up to 10 years of market exclusivity post-approval [2][6]
Sanofi Gets FDA Nod for Wayrilz in Immune Thrombocytopenia
ZACKS· 2025-09-01 17:31
Core Insights - Sanofi has received FDA approval for Wayrilz (rilzabrutinib), marking it as the first BTK inhibitor approved for treating persistent or chronic immune thrombocytopenia (ITP) in the United States [1][7] - The approval was based on the phase III LUNA 3 study, which demonstrated positive outcomes in sustained platelet counts and ITP symptoms, achieving both primary and secondary endpoints [2][7] - Wayrilz is also under review in the EU and China for ITP treatment and has been recently approved in the UAE for the same indication [3] Company Developments - Patients using Wayrilz will benefit from Sanofi's HemAssist program, which offers support for treatments related to rare blood disorders [3] - In addition to ITP, Wayrilz is being developed for other rare diseases, including IgG4-related disease (IgG4-RD) and warm autoimmune hemolytic anemia (wAIHA), with orphan drug designations from the FDA [5][9] - A phase II study is currently evaluating Wayrilz for IgG4-RD, a chronic immune-mediated condition affecting multiple organs [8] Stock Performance - Sanofi's shares have increased by 2.6% this year, contrasting with a 0.6% decline in the industry [4]
Press Release: Sanofi’s SAR446523, a GPRC5D monoclonal antibody, earns orphan drug designation in the US for multiple myeloma
Globenewswire· 2025-07-30 05:00
Core Viewpoint - Sanofi's SAR446523, a GPRC5D monoclonal antibody, has received orphan drug designation from the FDA for the treatment of relapsed or refractory multiple myeloma, highlighting the company's commitment to developing innovative therapies for this rare disease [1][2]. Group 1: Product Information - SAR446523 is an investigational IgG1-based monoclonal antibody designed to target GPRC5D, which is highly expressed on plasma cells, and aims to enhance antibody-dependent cellular cytotoxicity [3]. - The drug is currently undergoing a phase 1 clinical study in patients with relapsed or refractory multiple myeloma, with the clinical study identifier NCT06630806 [3]. Group 2: Disease Context - Multiple myeloma is the second most common hematologic malignancy, with over 180,000 new diagnoses globally each year, yet it remains incurable with a five-year survival rate of approximately 62% for newly diagnosed patients [4]. - There is a significant need for new therapeutic options, particularly for patients who are ineligible for transplants, due to high attrition rates in subsequent lines of therapy [4]. Group 3: Company Commitment - Sanofi is dedicated to advancing oncology treatments and aims to transform cancer care through the development of innovative therapies for rare and difficult-to-treat cancers [5]. - The company emphasizes its commitment to addressing urgent healthcare challenges and improving the lives of patients through its research and development efforts [6].
Press Release: Sanofi's SAR446523, a GPRC5D monoclonal antibody, earns orphan drug designation in the US for multiple myeloma
GlobeNewswire News Room· 2025-07-30 05:00
Core Viewpoint - Sanofi's SAR446523, a GPRC5D monoclonal antibody, has received orphan drug designation from the FDA for the treatment of relapsed or refractory multiple myeloma, highlighting the company's commitment to developing innovative therapies for this rare disease [1][2]. Group 1: Product Information - SAR446523 is an investigational IgG1-based monoclonal antibody designed to target GPRC5D, which is highly expressed on plasma cells, and aims to enhance antibody-dependent cell-mediated cytotoxicity [3]. - The drug is currently undergoing a phase 1 clinical study in patients with relapsed or refractory multiple myeloma, with the clinical study identifier NCT06630806 [3]. Group 2: Disease Context - Multiple myeloma is the second most common hematologic malignancy, with over 180,000 new diagnoses globally each year, yet it remains incurable with a five-year survival rate of approximately 62% for newly diagnosed patients [4]. - There is a significant need for new therapeutic options, particularly for patients who are ineligible for transplants, due to high attrition rates in subsequent lines of therapy [4]. Group 3: Company Commitment - Sanofi is dedicated to advancing oncology treatments and aims to transform cancer care through innovative therapies for rare and difficult-to-treat cancers [5]. - The company emphasizes its long-standing commitment to oncology and the development of first and best-in-class immunological and targeted therapies [5][6].
Qualigen Therapeutics Announces $4.5 Million Private Placement of Series A-3 Convertible Preferred Stock
Globenewswire· 2025-07-28 16:41
Core Points - Qualigen Therapeutics, Inc. has entered into definitive securities purchase agreements for the sale of Series A-3 Preferred Stock, raising a total of $4.5 million through the issuance of 4,500 shares at a stated value of $1,000 per share [1][2] - The Series A-3 Preferred Stock is convertible into 1,607,143 shares of common stock at a conversion price of $2.80 per share, subject to adjustments [1] - The net proceeds from this private placement will be used for working capital purposes [2] Regulatory and Compliance - The Series A-3 Preferred Stock was offered in a private placement under Section 4(a)(2) and/or Rule 506(b) of Regulation D under the Securities Act of 1933 and has not been registered under the Securities Act or applicable state securities laws [3] - The company has agreed to file a registration statement with the SEC covering the resale of the common stock underlying the Series A-3 Preferred Stock within 45 days after the closing date [4] Company Overview - Qualigen Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing novel therapeutics for cancer and infectious diseases, with a pipeline that includes QN-302, QN-247, and small-molecule RAS oncogene inhibitors [6]
Jaguar Health (JAGX) Earnings Call Presentation
2025-07-03 07:05
Clinical Development & Regulatory - Initial proof-of-concept results show crofelemer reduced Total Parenteral Nutrition (TPN) in patients with MVID and SBS-IF by up to 27% and 12.5% respectively[20], potentially modifying disease progression in intestinal failure patients[17] - Crofelemer achieved statistically significant improvement in monthly responder analysis in a prespecified subgroup of adult patients with breast cancer from the OnTarget Phase 3 study[23] - The company plans to meet with the FDA in Q2 2025 to discuss submitting a supplemental NDA for crofelemer for adult breast cancer patients on targeted therapy[21] - Napo Therapeutics is pursuing accelerated marketing PRIME authorization from the European Medicines Agency (EMA) for TPN reduction for MVID, as there are no alternative treatments[67] Commercial & Financial - Jaguar Health is a commercial-stage company with approximately $12 million in annual net revenue[13] - The company estimates the U S market revenue potential for Mytesi for HIV-related diarrhea to be approximately $30-$50 million in gross annual sales[27] - The global Short Bowel Syndrome (SBS) market is projected to reach $4.6 billion by 2027, with a CAGR of 26% from 2020 to 2027[27] - Net Q1 2025 revenue was approximately $2.2 million, a decrease of approximately 6% versus net Q1 2024 revenue of approximately $2.4 million and 37% versus net Q4 2024 revenue of approximately $3.5 million[110] Strategic Initiatives - Magdalena Biosciences, a joint venture focused on mental health illnesses, is valued at $5 million based on initial funding of $1 million from One Small Planet[103] - The company plans to pursue a Priority Review Voucher (PRV) for NP-300, with PRVs having sold for values ranging from $67 million to $350 million in past transactions[10, 97]
Press Release: Rilzabrutinib granted orphan drug designation in the US for sickle cell disease
Globenewswire· 2025-06-03 05:00
Core Viewpoint - The FDA has granted orphan drug designation to rilzabrutinib for sickle cell disease, highlighting its potential to address unmet medical needs in rare diseases [1][2]. Company Overview - Sanofi is an R&D driven biopharma company focused on improving lives through innovative medicines and vaccines, with a commitment to addressing urgent healthcare challenges [7]. Product Information - Rilzabrutinib is a novel, advanced, oral, reversible Bruton's tyrosine kinase (BTK) inhibitor that aims to restore immune balance through multi-immune modulation [5]. - The drug has received multiple orphan drug designations, including for immune thrombocytopenia (ITP), warm autoimmune hemolytic anemia (wAIHA), and IgG4-related disease (IgG4-RD) [2][8]. Disease Context - Sickle cell disease affects over 100,000 people in the US, predominantly impacting African American and Hispanic populations, leading to severe pain and reduced life expectancy [6]. - The disease is characterized by misshapen red blood cells that block blood flow, causing various health complications [6]. Regulatory Status - Rilzabrutinib is currently under regulatory review in the US, EU, and China for its potential use in ITP, with a target action date for FDA decision set for August 29, 2025 [3][8].
AB Science announces that EMA granted orphan drug status to AB8939 for the treatment of acute myeloid leukemia
Globenewswire· 2025-04-23 16:59
Core Viewpoint - The European Medicines Agency (EMA) has granted orphan drug designation to AB8939 for the treatment of acute myeloid leukemia (AML), indicating its potential significant benefit over existing therapies [1][2]. Group 1: Orphan Drug Designation - AB8939 previously received orphan drug designation from the US FDA, and the EU designation is a significant milestone, suggesting that it offers substantial benefits to AML patients [2]. - The criteria for orphan drug designation at EMA are stringent, requiring evidence of significant benefit compared to existing treatments [2][4]. Group 2: Efficacy and Safety Data - Preclinical data from mouse models show that AB8939 provides a significant benefit over current therapies like cytarabine, azacitidine, and venetoclax [3]. - Preliminary efficacy and safety data from Phase 1 trials indicate that AB8939 is effective as a monotherapy with different treatment cycles [3]. - AB8939 demonstrates efficacy against drug-resistant AML cells, with 45% of vincristine-resistant and 66% of cytarabine-resistant cells responding to treatment [4]. Group 3: Mechanism of Action - AB8939 is a synthetic molecule that targets cancer cells by destabilizing microtubules essential for cell division and inhibiting enzymes crucial for cancer stem cell survival [6]. - The chemical name of AB8939 is '1-{4-[2-(5-ethoxymethyl-2-methylphenylamino)-oxazol-5-yl]phenyl}imidazolidin-2-one' [6]. Group 4: Benefits of Orphan Drug Designation - Orphan drug designation in the EU provides various benefits, including scientific advice on product development, access to centralized marketing authorization, and potential financial incentives [8]. - If approved, AB8939 will enjoy 10 years of marketing exclusivity from the date of registration, provided the orphan designation remains valid [8].
Scilex Holding Company Announces FDA Has Approved Our Request for the Orphan Drug Designation for Colchicine for the Treatment of Pericarditis
Globenewswire· 2025-04-16 18:12
Core Insights - Scilex Holding Company has received FDA approval for Orphan drug designation for colchicine to treat pericarditis, enhancing its portfolio in non-opioid pain management products [1][3]. Company Overview - Scilex Holding Company focuses on acquiring, developing, and commercializing non-opioid pain management products for acute and chronic pain, with a commitment to improving patient outcomes [5][10]. - The company is headquartered in Palo Alto, California [9][11]. Product Portfolio - Scilex's commercial products include: - ZTlido (lidocaine topical system) 1.8% for neuropathic pain relief [5][7]. - ELYXYB, an oral solution for acute migraine treatment [5][7]. - Gloperba, the first liquid oral version of colchicine for gout flare prophylaxis [6][7]. - The company has three product candidates in development: - SP-102 (SEMDEXA™), a viscous gel for epidural injections targeting lumbosacral radicular pain, which has completed Phase 3 studies [8]. - SP-103, a next-generation lidocaine topical system for acute pain, recently completing Phase 2 trials [8]. - SP-104, a low-dose naltrexone hydrochloride for fibromyalgia treatment [8]. Regulatory Insights - The Orphan Drug Act allows the FDA to grant orphan designation to drugs intended for rare diseases, which can lead to seven years of market exclusivity upon first approval [3].