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Ctexli’s approval further entrenches Mirum as a leader in rare liver diseases, alongside its cornerstone product Livmarli and upcoming drug volixibat.

Drug development powered by artificial intelligence is countering incentives from the Inflation Reduction Act and making small molecules more attractive in the complex inflammatory & immunology disease space.

Around 300 FDA staffers laid off last week are being asked to return. So far, the Trump administration has terminated some 1,000 employees from the agency.

Non-opioid pain therapies are entering an unprecedented era, marked by the landmark FDA approval of Vertex’s Journavx and a growing number of alternative approaches. Their ultimate uptake, however, remains to be seen.

With the modality now in early clinical trials, experts say more efficiency, broader editing capabilities and delivery breakthroughs are needed to propel RNA editing to the next stage.

The proposed acquisition by global investment firms Carlyle and SK Capital Partners could net shareholders $3 per share plus potential CVR dollars and provide bluebird bio with primary capital to expand the commercial reach of its gene therapies.

Acelyrin earlier this month agreed to an all-stock merger with fellow immune player Alumis, with their combined cash tiding the combined company over until 2027.

The move comes weeks after Pfizer terminated its partnership with Sangamo Therapeutics for another hemophilia gene therapy.

Many of these unlawful and unauthorized shipments were explicitly tagged for compounding, according to a new analysis. Separately, a group of state attorneys general has raised concerns about the unsafe GLP-1 drugs finding their way to American consumers.

The postponed ACIP meeting comes barely a week after Robert F. Kennedy, Jr. was confirmed as Secretary of Health and Human Services, despite controversy regarding his anti-vaccine history.

The meeting, which will include Pfizer CEO Albert Bourla as the newly named board of directors’ chair, will reportedly cover key topics for the lobbying group, including the Inflation Reduction Act.

BridgeBio beat investor expectations with 1,028 unique prescriptions for ATTR-CM therapy Attruby, setting the company up to beat a 2025 sales consensus of $86 million.

Expanded sweetener molecule development program aims to broaden access to plant-based sweetening solutions tailored to diverse customer food and beverage categories.

• $5.5 million financing that closed on April 2, 2026 together with $5.7 million in capital raised in Q1-2026, expected to provide Company with cash runway into 2027 • $4.5 million of accrued VAT refunds and R&D refundable tax credits from UK subsidiary begin maturing, including $1.9 million VAT cash refund expected in June 2026 • Successful EMA and Australia TGA (ATGA) meetings aligned around use of existing biomarker and pending release of 3-year overall survival data to support Conditional Marketing Authorisations (CMAs) for OST-HER2 in prevention or delay of recurrence in fully-resected, pulmonary metastatic osteosarcoma (“Metastatic Osteosarcoma”) • 2.5-year overall survival data expected to be released during ASCO 2026 • EMA and ATGA have aligned on key design aspects of confirmatory Phase 3 Metastatic Osteosarcoma protocol required to have commenced prior to grant of CMAs or FDA BLA, including alignment on 3-year overall survival as a primary clinical efficacy endpoint • Upcoming U.S. FDA Type C meeting and U.K. MHRA SAM meeting to align with EMA & ATGA prior to Australia Clinical Trial Notification submission for Phase 3, including alignment on 3-year overall survival as a primary clinical efficacy endpoint. U.S. FDA (“FDA”) Pre-BLA meeting to confirm alignment on surrogate and primary clinical efficacy endpoints with EMA/ATGA, including alignment on 3-year overall survival as a primary clinical efficacy endpoint, and seek decisions on rolling review acceptance and RMAT designation following the EMA and U.K. MHRA granting ATMP designation (EMA/MHRA RMAT equivalent) for OST-HER2 in Metastatic Osteosarcoma • OS Animal Health S-1 filing for proposed ‘Go-Public’ transaction and receipt of SEC comments