Scholar Rock Reports the US FDA’s BLA Submission for Apitegromab to Treat Spinal Muscular Atrophy (SMA)

Shots: The US FDA received BLA, plus a request for a priority review of apitegromab to improve motor function in pts with SMA who are on SMN-targeted therapy. The EMA’s MAA to be filed in Q1’25 The BLA was supported by the P-III (SAPPHIRE) & P-II (TOPAZ) trial, where SAPPHIRE met its 1EP of…

ByDipanshu DixitJanuary 30, 2025

NEWS

Vertex Receives the US FDA Approval for Journavx (Suzetrigine) to Treat Acute Pain

Shots: The FDA has approved Journavx (50mg; BID) to treat moderate-to-severe acute pain in adults JOURNAVX is an oral, non-opioid pain signal inhibitor that selectively targets NaV1.8 relative to other NaV channels The company is also testing suzetrigine for peripheral neuropathic pain (PNP), with ongoing P-III trial for diabetic neuropathy and plans for…

ByDipanshu DixitJanuary 30, 2025

NEWS

Amgen Receives EC’s Approval for Blincyto to Treat Philadelphia Chromosome-Negative CD19-Positive B-Cell Precursor Acute Lymphoblastic Leukemia (B-ALL)

Shots: The EC has approved Blincyto monotherapy as a part of consolidation therapy to treat 1L philadelphia chromosome-negative CD19+ B-ALL adults based on P-III (E1910) trial Blincyto + CT showed improved OS with 4.5yrs. median follow up vs CT alone, whereas 5-yrs. mFU demonstrated 82.4% vs 62.5% OS in 112 pts The trial was led by…

ByDipanshu DixitJanuary 30, 2025

NEWS

Life Spine Receives US FDA’s 510(k) Clearance for ProLift Pivot Expandable Spacer System

Shots: The US FDA has granted 510(k) clearance to Prolift Pivot expandable spacer system, expanding Life Spine’s Micro Invasive tech portfolio The system provides full articulation for precise spinal fusion, while reducing tissue disruption & nerve retraction, allowing surgeons to restore patient alignment with superior control & efficiency. It also features OSSEO-LOC surface tech that promotes osseointegration & long-term…

ByDipanshu DixitJanuary 30, 2025

NEWS

Roche Reports Topline Data from P-III (INAVO120) Trial of Itovebi (Inavolisib) to Treat HR-Positive Breast Cancer

Shots: The P-III (INAVO120) trial evaluated Itovebi + Ibrance + fulvestrant vs matching PBO in PIK3CA-mutated, HR+/HER2-, endocrine-resistant, 1L LA/M breast cancer pts (n=325); ongoing regulatory review in EU & other regions Study met its 2EP of significant OS demonstrating 57% reduction in risk of disease worsening or death (mPFS: 15 vs 7.3mos.; HR=0.43).…

ByDipanshu DixitJanuary 30, 2025

NEWS

The EMA Accepts MAA for ImmunityBio’s Anktiva to Treat BCG-Unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC)

Shots: The EMA has accepted MAA of Anktiva + Bacillus Calmette-Guérin (BCG) to treat BCG-unresponsive NMIBC with CIS, & with/without papillary tumors, based on an ongoing QUILT 3.032 study. Application covers 27 EU countries, plus Iceland, Norway & Liechtenstein, with submission also made to the UK's MHRA As of the data cut-off on Jul…

ByDipanshu DixitJanuary 29, 2025

NEWS

Dyne Therapeutics’ DYNE-101 Secures the US FDA’s Fast Track Designation for Treating DM1

Shots: The US FDA has granted FTD to DYNE-101 for treating myotonic dystrophy type 1 (DM1). US Accelerated Approval is anticipated to submit in H1’26 DYNE-101 is being tested in the global P-I/II ACHIEVE trial for DM1 DYNE-101 is an antisense oligonucleotide (ASO) linked to an antibody fragment (Fab) that targets the transferrin…

ByDipanshu DixitJanuary 27, 2025

INSIGHTS+

EMA Marketing Authorization of New Drugs in December 2024

Shots: The EMA’s CHMP has granted positive opinions to 5 Biologics and 4 New Chemical Entities in December 2024, leading to treatments for patients and advances in the healthcare industry The major highlighted drugs were Galderma’s Nemluvio to treat Prurigo Nodularis and Atopic Dermatitis PharmaShots has compiled a list of 9 drugs that have been granted positive…

ByDipanshu DixitJanuary 23, 2025

INSIGHTS+

Disease of the Month – Epilepsy

Shots: Epilepsy is a chronic, non-communicable brain condition that affects neurons and causes recurring seizures Our Disease of the Month report covers epidemiology, market size, current management strategies, emerging therapeutic options, and the role of patient advocacy groups. Additionally, we highlight the key industry players advancing treatments for epilepsy For a detailed analysis and tailored…

ByDipanshu DixitJanuary 21, 2025

NEWS

Fermion Technology and Simcere Pharma Join Hands to Develop FZ002-037 for Pain Management

Shots: Fermion & Simcere Pharma have entered into an exclusive license and collaboration agreement to develop FZ002-037 for pain management As per the agreement, Simcere secures exclusive rights to develop & commercialize FZ002-037 across Greater China (Mainland China, Hong Kong, Macau & Taiwan) in exchange for an undisclosed upfront, milestones and sales-based tiered royalties…

ByDipanshu DixitJanuary 20, 2025

Dipanshu Dixit

Scholar Rock Reports the US FDA’s BLA Submission for Apitegromab to Treat Spinal Muscular Atrophy (SMA)

Vertex Receives the US FDA Approval for Journavx (Suzetrigine) to Treat Acute Pain

Amgen Receives EC’s Approval for Blincyto to Treat Philadelphia Chromosome-Negative CD19-Positive B-Cell Precursor Acute Lymphoblastic Leukemia (B-ALL)

Life Spine Receives US FDA’s 510(k) Clearance for ProLift Pivot Expandable Spacer System

Roche Reports Topline Data from P-III (INAVO120) Trial of Itovebi (Inavolisib) to Treat HR-Positive Breast Cancer

The EMA Accepts MAA for ImmunityBio’s Anktiva to Treat BCG-Unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC)

Dyne Therapeutics’ DYNE-101 Secures the US FDA’s Fast Track Designation for Treating DM1

EMA Marketing Authorization of New Drugs in December 2024

Disease of the Month – Epilepsy

Fermion Technology and Simcere Pharma Join Hands to Develop FZ002-037 for Pain Management

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