Neurocrine Biosciences Initiates P-I Study of NBIP-01435 to Treat CAH

Neurocrine initiated a P-I first-in-human study of NBIP-01435 to assess safety, PK/PD, tolerability, and immunogenicity in healthy adults via SC injection for the potential treatment of congenital adrenal hyperplasia (CAH)NBIP-01435 (CRF1 antagonist) aims to improve androgen control and reduce glucocorticoid dosing. In Dec’24, the FDA approved Neurocrine's crinecerfont, an oral CRF1 antagonist, to treat…

ByPrince GiriJuly 1, 2025

News

Omeros Submits MAA to the EMA for Narsoplimab (OMS721) to Treat TA-TMA

SShots:Omeros submitted an MAA to the EMA for narsoplimab to treat TA-TMA, with data demonstrating a 61% response rate and a 3x improvement in OS vs matched external controls; over 130 pts treated under expanded access data were also includedEMA granted Narsoplimab ODD status, and CHMP review will start mid-July under centralized…

ByPrince GiriJune 30, 2025

News

UCB Reports the P-III Data of Fenfluramine in CDKL5 Deficiency Disorder (CDD) Patients

Shots:UCB reported the P-III study (n=87; ages 1-35yrs.) of adjunctive fenfluramine vs. PBO in CDKL5 Deficiency Disorder (CDD) diagnosis and uncontrolled seizure pts.The P-III study met its 1EP (median % reduction in countable motor seizure frequency) and most key 2EPs. Fenfluramine was well tolerated with a consistent safety profile in prior DS/LGS studiesUCB…

ByPrince GiriJune 30, 2025

VIEWPOINTS

Bringing Awareness to the Public: Samuel S. Gidding & Allison Jamison in an Enlightening Conversation with PharmaShots

Shots: Homozygous Familial Hypercholesterolemia (HoFH) is a rare genetic condition that affects approximately 1 in 250,000 Americans. It is characterized by the presence of two genetic mutations that lead to extremely high levels of LDL cholesterol. In this interview, Sam Gidding and Allison Jamison from the Family Heart Foundation/ emphasize the critical need for enhanced…

BySaurabh ChaubeyNovember 18, 2024

INSIGHTS+

Disease of the Month – Fabry Disease

Shots: Fabry disease is a rare inherited neurological disorder that affects the body's ability to break down lipids into smaller components In this reprise of our Disease of the Month report, we bring an illuminating account of Fabry Disease with deep dive analysis of epidemiology, market size, disease management, available therapies, patient advocacy groups, and…

BySenior EditorJune 12, 2024

INSIGHTS+

Disease of the Month – Sjögren’s Syndrome

Shots: A rare autoimmune condition, Sjögren’s Syndrome is characterized by dry mouth and eye. The condition affects 0.5 to 1% of the global population of which 90% of patients are women In this reprise of our Disease of the Month report, we bring an illuminating account of Sjögren’s Syndrome with deep dive analysis of epidemiology, market size, disease management, available therapies, patient…

BySaurabh ChaubeyMay 16, 2024

VIEWPOINTS

Breaking the Barriers of hATTR Treatment: Thirusha Lane Shares her Insightful Thoughts with PharmaShots

Shots: Often, patient care focuses solely on therapeutics, but with rare diseases like hATTR amyloidosis, integrating holistic care for patients is crucial to ensure patients fully live their lives In an enlightening conversation with PharmaShots, Thirusha Lane shares the highlights of the recommendations developed by the international panel of patient advocates and HCPs for people…

BySaurabh ChaubeyJanuary 22, 2024

EXCLUSIVE

Insights from Coralie Gressier-Sayag on Avapritinib Efficacy Data Presented at EHA 2023

Shots:Coralie started by giving a brief introduction about Systemic Mastocytosis (SM), a rare disease. She told us that in Europe, approximately 40,000 people live with SM, with 80-90% living with Indolent SM and the rest with Advanced SMShe then gave an overview of the P-II PATHFINDER study evaluating avapritinib. The study's 2-year follow-up…

BySenior EditorAugust 30, 2023

THOUGHTSPOT

Getting on with Rare Disease Therapeutic Options (Orphan Disease Therapies)

Shots:With the healthcare sector moving ahead with its technological advances, there is still an unexplored section in therapeutics that requires much more attention. Through innovations, life science companies have evolved to make breakthrough discoveries in many focus areas, but when it comes to the rare disease segment, all our endeavors seem to be bleak…

BySaurabh ChaubeyMarch 14, 2023

VIEWPOINTS

PharmaShots Interview: Abdul Mullick, CEO at Kyowa Kirin Shares Insights on the Launch of a Virtual Exhibition to Highlight Issues of Rare Disease, X-linked Hypophosphataemia

Shots:Abdul spoke about the launch of a digital exhibition ‘Shine a light’ to raise awareness about rare diseasesAbdul also emphasized the significance of this awareness campaign for the patients who were unaware of X-linked Hypophosphataemia(XLH)The interview provides a profound understanding of Kyowa Kirin's initiatives to help patients suffering from XLHSmriti: Can we…

BySenior EditorJuly 27, 2022

Neurocrine Biosciences Initiates P-I Study of NBIP-01435 to Treat CAH

Omeros Submits MAA to the EMA for Narsoplimab (OMS721) to Treat TA-TMA

UCB Reports the P-III Data of Fenfluramine in CDKL5 Deficiency Disorder (CDD) Patients

Bringing Awareness to the Public: Samuel S. Gidding & Allison Jamison in an Enlightening Conversation with PharmaShots

Disease of the Month – Fabry Disease

Disease of the Month – Sjögren’s Syndrome

Insights from Coralie Gressier-Sayag on Avapritinib Efficacy Data Presented at EHA 2023

Getting on with Rare Disease Therapeutic Options (Orphan Disease Therapies)

PharmaShots Interview: Abdul Mullick, CEO at Kyowa Kirin Shares Insights on the Launch of a Virtual Exhibition to Highlight Issues of Rare Disease, X-linked Hypophosphataemia

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