Drugs

Rare Disease Innovation Hub 2026 Strategic Agenda

Having operated since its Fall 2024 inception with no dedicated budget and only the director as a full-time staff member, the FDA Rare Disease Innovation Hub is poised to move forward in 2026 with $1 million in funds from CBER and CDER and a small dedicated staff. The Hub is a point of collaboration and connectivity between the agency’s medical product Centers and rare disease programs to improve outcomes for rare disease patients.

Its stated goals are to:

further advance the regulatory science of rare disease therapies;
enhance and strengthen coordination and alignment between medical product Centers; and
create a centralized point of contact for external partners.

The Hub’s Strategic Agenda 2026 projects these 2026 actions:

rare disease innovation, science, and exploration workshop series;
patient and patient organization engagement in the development and review of drugs for rare diseases;
promote knowledge-sharing between medical product Centers on rare disease treatment review;
rare disease review staff training feedback pilot;
public, agency-wide rare disease newsletter;
website-based rare disease roadmap;
hosting FDA Rare Disease Day 2026; and
encourage Hub interaction with the community at multiple meetings and conferences.

Gupta Raises Concerns About Overton’s Independence, Ideology

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FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

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FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome