Pink Sheet - News And Expert Analysis On Pharma Policy And Regulation since 1939

Regulatory Trackers


US FDA Performance Trackers

US FDA Performance Trackers

EU Performance Trackers

EU Performance Trackers

Pink Sheet Global Guidance Tracker

Global Guidance Tracker

The Rapid Changes To Accelerated Approvals


Pathways & Standards


Could Another US FDA Safety Alert Foreshadow Withdrawal Of Intercept’s Ocaliva?

 
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The FDA's view that current monitoring recommendations may not be sufficient to mitigate harm increases the likelihood it could seek withdrawal.

Real-World Evidence Helps Open Accelerated Approval Pathway For Neurodegeneration Therapies

 

Clene’s ALS drug CNM-Au8 and UniQure’s gene therapy for Huntington’s disease emerged from US FDA discussions with accelerated approval plans using real-world evidence from compassionate use and natural history data sources.

AstraZeneca Sees Bright Future For Registry-Based Randomized Clinical Trials

 

AZ’s DAPA-MI trial showed the feasibility of R-RCT designs to support new indications in drug development and more established uses to inform medical practice.

Signal Boost: Real-World Evidence Fits US FDA’s Safety Ambitions More Easily Than Efficacy

 

RWE is already transforming the US FDA’s active surveillance systems and could catalyze change in pregnancy postmarketing studies, but is advancing more slowly for use as efficacy evidence.

Regional Comparisons


How The UK, EU, Canada, Australia And Japan Are Tackling Diversity In Clinical Trials

 
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As efforts to improve diversity in clinical trials gain momentum globally, the Pink Sheet asked regulators in the UK, the EU, Canada, Australia and Japan about their efforts to support representative enrolment. 

EU Revokes Intercept/Advanz’s Ocaliva Approval As Moment Of Reckoning Nears in US

 
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The EU marketing authorization for the primary biliary cholangitis treatment has now been revoked. Meanwhile the drug's approval is in jeopardy in the US, where an advisory committee will opine on whether the accelerated approval drug has confirmed clinical benefit.

England Lags Behind Eight European Countries On Innovative Drug Availability

 

In this third article of a series on new drug reimbursement recommendations by the HTA body NICE, the Pink Sheet finds that fewer innovative medicines are reimbursed in England than in eight other European nations.

Decoding Regulatory Agency Decisions: The Case of Amylyx’s Relyvrio/Albrioza In The US And EU

 

Relyvrio/Albrioza, Amylyx’s ill-fated treatment for amyotrophic lateral sclerosis, was approved for marketing by the US Food and Drug Administration but rejected by the European Medicines Agency. The Pink Sheet explores how the two agencies applied the available regulatory flexibilities and the impact of other factors, such as regulatory precedence and patient influence, on decision-making.

Drug Review Profiles


US FDA Review Of Akebia’s Vafseo Informed By Roxadustat, Daprodustat Safety Concerns

 
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Vadadustat's review overlapped with two other oral HIF-PH class drugs. Safety issues with FibroGen/AstraZeneca’s roxadustat drew attention during vadadustat's first-cycle review, while GSK’s Jesduvroq labeling and postmarketing requirements informed the second-cycle approval.

Akebia’s Vafseo: Japanese Postmarketing Data, Narrowed Indication Eased Liver Toxicity Worries

 
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The Pink Sheet Drug Review Profile explores the US FDA’s approval of vadadustat to treat anemia in chronic kidney disease patients on dialysis. A complete response letter cited the risk of drug-induced liver injury, but postmarketing data from Japan reassured reviewers.

Vafseo Chronology: Complete Response Letter, Dispute Resolution And A Second-Cycle Approval

 
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The Pink Sheet’s Drug Review Profile looks at the timeline behind the development and US FDA approval of Akebia’s Vafseo, a treatment for anemia in chronic kidney disease patients on dialysis.

Biosimilars: US FDA’s Updated Scientific Thinking Led To Interchangeability For Amgen's Wezlana

 
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Pink Sheet’s Drug Review Profile explores Amgen’s Wezlana, an interchangeable biosimilar to Janssen’s Stelara. FDA determined late in the review that switching studies generally would not be needed for interchangeable ustekinumab products, prompting Amgen to seek the designation.

Interviews


Wider Gene Therapy Definition In EU Will Require ‘Myth-Busting’ From EMA

 

The European Commission’s proposed pharma legislation overhaul could expand the definition of a gene therapy medicinal product, posing challenges both from a regulatory and public perception perspective.

EURORDIS Chief Backs ‘Collective Bargaining’ For Orphan Drugs Under EU Pharma Reform

 

The European Parliament proposed introducing joint procurement for orphan medicinal products in the EU pharma legislation overhaul – a move that the new CEO of EURORDIS says could lead to “faster and more equitable” access to drugs for rare diseases.

KRSC Aims To Raise Korea’s Regulatory Science Game

 
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The head of the Korea Regulatory Science Center talks exclusively to the Pink Sheet about why regulatory science is crucial, the country's efforts to improve in the area and the center's strategic bridging role.

EU HTA Regulation A Positive Move For Rare Disease Therapies, says EURORDIS Chief

 

Greater transparency around EU health technology assessment processes “can only be a good thing” for innovative rare disease therapies, Virginie Bros-Facer, the new CEO of the EU network of rare disease patient organizations, EURORDIS, tells the Pink Sheet.