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NEWS

Glaukos Receives FDA Approval for Epioxa

Shots:Glaukos reported the US FDA approval of Epioxa HD/Epioxa, an incision-free, topical drug therapy for keratoconus, offering a non-surgical alternative to traditional corneal cross-linkingThe approval was based on two P-III pivotal studies (>400 pts) that met its pre-specified 1EPs strong efficacy and safety; Epioxa uses a UV-activated, oxygen-enriched formulation that preserves the corneal…
October 22, 2025

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NEWS

VectorY Therapeutics Collaborates with Shape Therapeutics to Develop Vectorized Antibodies for Neurodegenerative Diseases

Shots:VectorY has entered into an option & license deal with Shape to assess Shape’s SHP-DB1 (AAV capsid) for vectorized Abs payloads against 3 targets & may secure an exclusive license for their delivery if evaluation succeedsAs per the deal, VectorY will handle development & commercialization of resulting therapies in exchange for an upfront…
September 18, 2025

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NEWS

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…
July 1, 2025

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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…
November 18, 2024

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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…
June 12, 2024

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Disease of the Month – Sjögren’s Syndrome
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…
May 16, 2024

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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…
January 22, 2024

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