Calcium

FDA Approves Belzutifan as First Oral Treatment for Advanced Pheochromocytoma and Paraganglioma

8 days ago

• The FDA has approved belzutifan (Welireg) for adult and pediatric patients 12 years and older with locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma, based on promising phase 2 trial results. • As a selective HIF-2α inhibitor, belzutifan demonstrated a 26% overall response rate with a median duration of response of 20.4 months, offering a novel mechanism of action compared to historically used treatments. • This approval is particularly significant as previous treatment options were limited, with the only previously approved agent no longer commercially available and alternative therapies associated with concerning toxicity profiles.

Clinical and Radiographic Evaluation of Silver Diamine Fluoride Versus Mineral Trioxide Aggregate in Indirect Pulp Capping

4 months ago

A randomized clinical trial compared the effectiveness of Silver Diamine Fluoride (SDF) and Mineral Trioxide Aggregate (MTA) in indirect pulp capping (IPC) for first permanent molars with deep carious lesions. Both materials showed a 100% clinical and radiographic success rate after 12 months, with no significant differences in dentin bridge thickness. The study highlights the potential of SDF as a cost-effective alternative to MTA for IPC in permanent teeth, despite the need for further research.

Rilzabrutinib Shows Promise in Phase 3 Trial for Immune Thrombocytopenia

5 months ago

• Rilzabrutinib demonstrated a durable platelet response in 23% of ITP patients, compared to 0% with placebo, meeting the primary endpoint of the LUNA 3 trial. • Patients receiving rilzabrutinib were approximately three times more likely to achieve a platelet response than those on placebo. • The trial also met key secondary endpoints, including reduced bleeding, decreased need for rescue therapy, and improved physical fatigue and quality of life. • Rilzabrutinib is currently under regulatory review in the US and the EU, with a target FDA action date of August 29, 2025.

Ascendis Pharma's Yorvipath Commercially Available in the US, Offering New Hope for Hypoparathyroidism

6 months ago

• Ascendis Pharma has launched Yorvipath (palopegteriparatide) in the US, the first FDA-approved treatment for hypoparathyroidism in adults. • Yorvipath is a once-daily prodrug of parathyroid hormone, designed to provide continuous exposure to active PTH over 24 hours. • Ascendis has established a dedicated support program to assist patients with clinical education, access, and injection training. • Clinical trials demonstrated that 93% of YORVIPATH-treated patients achieved independence from both active vitamin D and therapeutic doses of elemental calcium.

Milestone Pharmaceuticals Gains New Patent for Etripamil, Prepares for Potential Launch

6 months ago

• Milestone Pharmaceuticals received a Notice of Allowance from the USPTO for a new patent covering the repeat dose regimen of etripamil nasal spray, extending potential IP protection to July 2042. • The patent supports the New Drug Application (NDA) for CARDAMYST™ (etripamil) currently under FDA review, with a PDUFA target date of March 27, 2025. • Milestone is actively preparing for a mid-2025 commercial launch of CARDAMYST for Paroxysmal Supraventricular Tachycardia (PSVT), pending FDA approval. • A Phase 3 trial of etripamil nasal spray for Atrial Fibrillation with Rapid Ventricular Rate (AFib-RVR) is planned to commence in the first half of 2025.

Cytokinetics Initiates Phase 1 Trial of CK-4015089 for Muscle Disorders

6 months ago

• Cytokinetics has begun a Phase 1 clinical trial for CK-4015089, a fast skeletal muscle troponin activator (FSTA). • The study will assess the safety, tolerability, and pharmacokinetics of CK-089 in healthy participants through single and multiple ascending doses. • Preclinical data showed CK-089 improved muscle force and function in animal models, suggesting potential for treating muscular dystrophy. • The trial represents Cytokinetics' expansion into neuromuscular diseases, while the company's cardiology franchise remains the priority.

CAVALIER Trial: Early Intervention for Trauma Patients with Calcium and Vasopressin

6 months ago

• Hennepin Healthcare is seeking approval to enroll trauma patients in the CAVALIER trial without prior consent, focusing on early blood loss treatment. • The CAVALIER trial investigates whether administering calcium and vasopressin earlier improves survival rates in trauma patients. • The study is part of the LITES Network, aiming to enroll over 1,000 adult trauma victims across multiple sites. • This initiative follows previous controversies, emphasizing transparency and ethical considerations in emergency research.

Hennepin Healthcare Seeks Exception for Trauma Study on Blood Loss Treatment

6 months ago

• Hennepin Healthcare is seeking an exception to enroll unconscious trauma patients in a clinical trial without prior consent to study early interventions for blood loss. • The study will investigate whether early administration of calcium and vasopressin, compared to a placebo, improves survival rates in trauma patients. • The LITES Network-affiliated trial aims to enroll over 1,000 adult trauma victims nationally, with Hennepin Healthcare participating as one arm. • The hospital is engaging with the community to build trust and ensure ethical oversight following previous concerns about patient consent in research.

JMT103 Demonstrates Efficacy in Unresectable Giant Cell Tumor of Bone

7 months ago

• JMT103, a novel therapy, shows promising efficacy in treating unresectable or surgically-challenging giant cell tumor of bone (GCTB). • The Phase Ib/II study demonstrated a notable objective tumor response rate (OTR) based on histopathological or radiological evaluation. • Treatment with JMT103 led to clinically meaningful reductions in pain and improvements in daily functioning for GCTB patients. • The safety profile of JMT103 was manageable, with adverse events consistent with the mechanism of action and manageable with supportive care.

FDA Accepts Milestone's Cardamyst NDA for PSVT Treatment

a year ago

• The FDA has accepted Milestone Pharmaceuticals' NDA for Cardamyst (etripamil) nasal spray, a potential treatment for paroxysmal supraventricular tachycardia (PSVT). • Cardamyst is designed for patient self-administration, offering a rapid-response therapy to manage PSVT episodes without immediate medical oversight. • The PDUFA target date for the FDA's decision on Cardamyst is set for 10 months from the NDA acceptance date of May 26, 2024. • PSVT affects an estimated two million people in the U.S., and Cardamyst aims to provide a convenient and effective treatment option.

Milestone Pharmaceuticals Resubmits NDA for Etripamil Nasal Spray for PSVT Treatment

a year ago

• Milestone Pharmaceuticals has resubmitted its New Drug Application (NDA) to the FDA for etripamil nasal spray, a potential treatment for paroxysmal supraventricular tachycardia (PSVT). • The resubmission addresses issues raised in a previous Refusal to File letter from the FDA, incorporating restructured data sets and reformatted data files. • Etripamil is designed as a self-administered rapid response therapy, offering patients a new option for on-demand management of PSVT episodes. • If approved, etripamil nasal spray could provide a valuable treatment option for the estimated two million people in the U.S. diagnosed with PSVT.

Drug Development Updates