BioSpace News Archive

FDA

Mirum Secures Livmarli’s Expansion into Familial Intrahepatic Cholestasis

Wednesday’s FDA approval expands Mirum’s Livmarli into the rare genetic disorder that causes progressive liver disease. The biotech has also filed a supplemental New Drug Application for a higher dose of the drug and allowing its use in younger patients.

March 14, 2024
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2 min read
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Tristan Manalac
Drug Delivery

The Unintended Consequences of Small Molecules

This episode focuses on a healthy discussion regarding the IRA, particularly the unintended consequences to small molecule development within the industry and for patients.

March 14, 2024
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1 min read
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Lori Ellis
Pharm Country

Merck to Start Trials of New HPV Vaccine and Single-Dose Gardasil Shot

Merck is looking to expand its offerings in the human papillomavirus space with a new vaccine and to study a single-shot variant of Gardasil 9, both of which will start trials toward the end of 2024.

March 14, 2024
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2 min read
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Tyler Patchen
Policy

FDA Approves Madrigal’s Rezdiffra as First MASH Therapy

Madrigal Pharmaceuticals’ Rezdiffra (resmetirom) is the first-ever approved therapy for metabolic dysfunction-associated steatohepatitis—a decision experts say could signal a sea change in treatment of the disease.

March 14, 2024
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6 min read
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Jill Neimark
Policy

PhRMA Loses Another Legal Battle as Court Sides with Arkansas Over Drug Discount Program

A lawsuit filed by the Pharmaceutical Research and Manufacturers of America failed to block an Arkansas law that empowers hospitals to use outside pharmacies to dispense discounted drugs.

March 14, 2024
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2 min read
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Tristan Manalac
Deals

AstraZeneca Boosts Rare Disease Portfolio with $1.05B Amolyt Acquisition

At the center of the deal is Amolyt Pharma’s late-stage candidate eneboparatide for the rare disease hypoparathyroidism. AstraZeneca also gains ownership of AZP-3813, which is being assessed for acromegaly in a Phase I trial.

March 14, 2024
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2 min read
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Tristan Manalac
ADC Biotech Tubulis Closes Upsized $138M Financing Round

The German biotech Thursday said the proceeds from the Series B2 round will support clinical evaluation of its lead solid tumor antibody-drug conjugate candidates and expansion into the U.S.

March 14, 2024
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2 min read
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Tyler Patchen
Policy

Vertex Fails to Convince UK Watchdog of CRISPR Therapy Casgevy’s Value

The U.K. National Institute for Health and Care Excellence on Thursday recommended against funding Vertex Pharmaceuticals’ CRISPR-based sickle cell disease therapy Casgevy unless uncertainties can be cleared up.

March 14, 2024
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2 min read
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Nick Paul Taylor
Policy

BIO Separates from China’s WuXi AppTec Amid National Security Concerns

In a change of position as congressional scrutiny of WuXi AppTec grows, the Biotechnology Innovation Organization announced it is taking steps to separate from the China-based biotech.

March 14, 2024
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3 min read
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Tristan Manalac
Innate Pharma Announces Conference Call and Webcast for Full Year 2023 Financial Results

Regulatory News: Innate Pharma SA, announced that the Company will hold a conference call on Thursday, March 21, 2024 at 2 p.m. CET / 9 a.m. EDT, following the release of its financial results for the full year ending December 31, 2023.

March 14, 2024
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3 min read