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NEWS

Ultragenyx Reports the EC’s Approval of Evkeeza (evinacumab) to Treat 6-Months Old Children with Homozygous Familial Hypercholesterolemia (HoFH)

Shots: The EC has approved Evkeeza (an angiopoietin-like 3 inhibitor), as an adjunct to diet & other lipid-lowering therapies, to treat children (6mos. to 5yrs.) with HoFH Approval was based on model-based analysis that predicted similar or greater LDL-C reductions with Evkeeza (15mg/kg, Q4W) in children (6mos. to 5yrs.) at 24wks. Data from compassionate…
January 6, 2025

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NEWS

NMD Pharma’s NMD670 Secures the US FDA’s Orphan Drug Designation to Treat Charcot-Marie-Tooth Disease

Shots: The US FDA has granted ODD to NMD670 for treating Charcot-Marie-Tooth disease (CMT). It is small molecule that inhibits CIC-1 to enhances muscle responsiveness, improving neuromuscular transmission & restoring function NMD670 (PO, BID) is under P-IIa (SYNAPSE-CMT) evaluation for its efficacy, safety & tolerability in adults (n=80) with CMT1 or CMT2 subtype for over…
January 6, 2025

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Astellas
NEWS

Astellas’ Vyloy (Zolbetuximab) Secures the NMPA’s Approval as a 1L Treatment of Advanced G/GEJ Adenocarcinoma

Shots: The NMPA has granted approval to Vyloy + fluoropyrimidine & Pt-based CT as a 1L treatment of locally advanced unresectable or metastatic HER2-, CLDN18.2+ G/GEJ adenocarcinoma Approval was supported by P-III (GLOW: n=145 & SPOTLIGHT: n=36) studies assessing Vyloy + CAPOX & mFOLFOX6, respectively, vs PBO among Chinese patients with this indication …
January 6, 2025

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WEEKLY SNAPSHOT

PharmaShots Weekly Snapshots (December 30th, 2024 – January 03rd, 2025)

This week PharmaShots’ news was all about the updates on Clinical Trials, Regulatory, Pharma, MedTech, M&A, Biosimilars, Animal Health & Biotech. Check out our full report below:     Novartis Reports Data from P-III (STEER) Study of Onasemnogene Abeparvovec to Treat Children and Young Adults with Spinal Muscular Atrophy (SMA) Read More: Novartis CARsgen Therapeutics Reports Topline Results from P-II…
January 3, 2025

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GSK
NEWS

GSK’s Nucala (Mepolizumab) Secures the NMPA’s Approval for Treating Chronic Rhinosinusitis with Nasal Polyps (CRSwNP)

Shots: The NMPA has approved Nucala as an add-on therapy with intranasal corticosteroids in CRSwNP adults with inadequately controlled disease. It is approved for patients (≥12yrs.) as an add-on treatment of severe eosinophilic asthma & for adults having eosinophilic granulomatosis with polyangiitis Approval was based on 2 P-III studies: MERIT assessing Nucala vs PBO…
January 3, 2025

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Innovent
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Innovent Reports the NMPA’s Approval of Dovbleron (Taletrectinib Adipate Capsule) for Treating ROS1-Positive NSCLC

Shots: The NMPA has approved another NDA of Dovbleron (ROS1 tyrosine kinase inhibitor) for treating locally advanced or metastatic ROS1+ NSCLC. It was approved for those with ROS1+ NSCLC previously treated with ROS1 TKIs in Dec 2024 The approval was supported by pivotal P-II (TRUST-I) study assessing safety, tolerability and efficacy of Dovbleron to…
January 3, 2025

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NEWS

Chimerix Reports the Dordaviprone’s NDA Submission to the US FDA for Recurrent H3 K27M-Mutant Diffuse Glioma

Shots: Chimerix has submitted the NDA of dordaviprone (small molecule imipridone targeting the mitochondrial protease ClpP & dopamine receptor D2) to the US FDA, seeking accelerated approval for treating recurrent H3 K27M-mutant diffuse glioma Chimerix has also requested priority review along with a Rare Paediatric Disease PRV as a part of submission, as the…
January 2, 2025

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NEWS

MicuRx Pharmaceutical’s MRX-5 Secures the US FDA’s Orphan Drug Designation for Treating NTM Infections

Shots: The US FDA has granted orphan drug designation (ODD) to MRX-5 for treating non-tuberculous mycobacteria (NTM) infections MRX-5 is a new drug for drug-resistant NTM infections, showing strong antibacterial activity, favorable safety & PK profile in both animal studies & human trial. With minimal interactions, low resistance potential & high oral bioavailability, it…
January 2, 2025

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Hutchmed
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HUTCHMED Reports the NMPA’s NDA Acceptance of Orpathys Plus Tagrisso with Priority Review for EGFR-Mutated NSCLC with MET Amplification

Shots: The NMPA has accepted and granted priority review to NDA of Orpathys (savolitinib) + Tagrisso (osimertinib) for treating locally advanced or metastatic EGFR-mutated NSCLC with MET amplification in patients progressed on 1L EGFR inhibitor therapy. HUTCHMED will receive milestone from AstraZeneca Submission was based on P-III (SACHI) study assessing the safety & efficacy…
January 2, 2025

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NEWS

Corcept Therapeutics Reports NDA Submission of Relacorilant to the US FDA for Treating Hypercortisolism

Shots: Corcept has submitted NDA of relacorilant to the US FDA for the treatment of endogenous hypercortisolism (Cushing’s syndrome) Submission was based on pivotal GRACE study, confirmatory P-III (GRADIENT) & long-term extension trials plus P-II hypercortisolism trial. Relacorilant showed improved symptoms without adrenal insufficiency, hypokalemia, QT prolongation, or progesterone receptor-related AEs (endometrial thickening or…
January 1, 2025

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