FDA Approves Tremfya for Crohn's Disease and Fabhalta for Rare Kidney Disease Amid Major Pharma Acquisitions
核心洞察
The FDA has approved Johnson & Johnson's Tremfya for moderately to severely active Crohn's disease (搜索), marking its fourth indication in the US based on robust data from phase III GALAXI and GRAVITI studies.
Novartis received FDA approval for Fabhalta to treat C3 glomerulopathy (搜索), becoming the first and only drug approved to address the underlying cause of this ultra-rare progressive kidney disease.
AstraZeneca announced plans to acquire Belgium-based EsoBiotec (搜索) for up to $1 billion, while Sanofi is purchasing Dren Bio (搜索)'s DR-0201 for $600 million upfront to strengthen its immunology pipeline.
The U.S. Food and Drug Administration (FDA) has approved two significant therapies this week, while major pharmaceutical companies announced strategic acquisitions to bolster their pipelines.
FDA Approves J&J's Tremfya for Crohn's Disease
The FDA has granted approval to Johnson & Johnson's IL-23 (搜索) inhibitor Tremfya (guselkumab) for the treatment of moderately to severely active Crohn's disease (搜索) (CD), marking its second inflammatory bowel disease (IBD) indication. The approval covers both subcutaneous (SC) and intravenous (IV) induction regimens.
The regulatory decision was supported by data from the phase III GALAXI and GRAVITI studies, which demonstrated Tremfya's robust efficacy in achieving clinical and endoscopic endpoints in CD patients.
Tremfya was previously approved for moderately to severely active ulcerative colitis in September 2024, and already holds approvals for moderate-to-severe plaque psoriasis and active psoriatic arthritis. This latest indication represents Tremfya's fourth approved use in the United States. The therapy is currently under regulatory review in the European Union for both ulcerative colitis and Crohn's disease (搜索) indications.
In related news, J&J announced that the FDA has granted Fast Track designation to nipocalimab, a key pipeline candidate, for treating moderate-to-severe Sjögren's disease (搜索) (SjD). This follows the Breakthrough Therapy Designation granted to nipocalimab for SjD in November 2024. The company is currently enrolling patients in the phase III DAFFODIL study after nipocalimab met the primary endpoint in the phase II DAHLIAS dose-ranging study earlier this year.
Novartis' Fabhalta Receives Approval for Rare Kidney Disease
The FDA has approved Novartis' Fabhalta (iptacopan) for the treatment of adults with C3 glomerulopathy (搜索) (C3G), an ultra-rare, progressive kidney disease. The approval was based on data from the APPEAR-C3G study.
With this approval, Fabhalta, an oral alternative complement pathway inhibitor, becomes the first and only drug approved to treat the underlying cause of C3G. Similar applications for Fabhalta in C3G are currently under review in the European Union, China, and Japan.
Fabhalta is already approved for treating paroxysmal nocturnal hemoglobinuria and for reducing the risk of rapid disease progression in proteinuria adults with primary IgA nephropathy. Novartis is evaluating the drug in a broad range of rare kidney diseases, including atypical hemolytic uremic syndrome, immune complex membranoproliferative glomerulonephritis, and lupus nephritis.
AstraZeneca to Acquire EsoBiotec for Up to $1 Billion
AstraZeneca has announced a definitive agreement to acquire Belgium-based EsoBiotec (搜索), a leading vivo cell therapy company, for a total consideration of up to $1 billion in cash on a debt-free basis. The acquisition will integrate EsoBiotec's proprietary ENaBL platform, which is used to develop transformative cell therapy treatments that can be delivered in minutes, in contrast to traditional cell therapies that take weeks to prepare. The transaction is expected to close in the second quarter of 2025.
In a separate deal, AstraZeneca is in-licensing worldwide rights to Alteogen's novel hyaluronidase enzyme, ALT-B4, for developing subcutaneous formulations of multiple oncology assets. ALT-B4 has been developed utilizing the Hybrozyme platform technology. Financial terms include an undisclosed upfront payment to Alteogen, with additional development, regulatory, and sales-related milestone payments.
The European Commission has also granted approval for the expanded use of AstraZeneca's blockbuster drug, Imfinzi (durvalumab), as a monotherapy for adults with limited-stage small cell lung cancer (搜索) whose disease has not progressed following platinum-based chemoradiation therapy. This approval was based on data from the phase III ADRIATIC study. The FDA approved Imfinzi for a similar indication in December 2024.
Additionally, AstraZeneca's pipeline candidate eneboparatide met its primary endpoint of normalizing serum calcium levels at 24 weeks with statistical significance in the phase III CALYPSO study. The study evaluated the drug in adults with chronic hypoparathyroidism (搜索), a rare endocrine disease. Eneboparatide was added to AstraZeneca's pipeline following the acquisition of Amolyt Pharma last year.
Sanofi Strengthens Immunology Pipeline with Dren Bio Acquisition
Sanofi is acquiring private biotech Dren Bio (搜索)'s targeted bispecific myeloid cell engager (MCE), DR-0201, for an upfront payment of $600 million. In clinical studies, DR-0201 has shown robust B-cell depletion, which is believed to have the potential to reset the adaptive immune system—a potential game-changer for treating autoimmune diseases, particularly for patients refractory to existing treatments.
DR-0201 is currently being evaluated in two phase I studies. In addition to the upfront payment, Dren Bio (搜索) will also be entitled to potential milestone payments of up to $1.3 billion. The transaction is expected to close in the second quarter of 2025.
Pfizer Divests Remaining Stake in Haleon
Pfizer has divested its remaining 7.3% stake in Haleon, approximately 662 million shares, to institutional investors and Haleon itself for £3.85 per ordinary share (around £2.5 billion). Pfizer sold 618 million ordinary shares to institutional investors worth around $3.1 billion (approximately £2.4 billion). Per a previous share buyback plan, Pfizer sold around 44.14 million shares worth approximately $220 million (£170 million) directly to Haleon.
Haleon was originally a consumer health joint venture created by Pfizer and GSK in 2019, with GSK owning a controlling stake of 68%. GSK/Pfizer divested the Consumer Healthcare JV to form Haleon in July 2022. GSK, which initially owned a nearly 13% stake in Haleon, sold its entire stake in May 2024. Pfizer originally held a 32% stake in Haleon and had been gradually reducing it since 2022.
Market Performance
The NYSE ARCA Pharmaceutical Index has risen 2.09% in the past five trading sessions. Among major pharmaceutical stocks, Eli Lilly rose the most (5.1%) over the last five trading sessions, while Johnson & Johnson, Merck, and AstraZeneca remained flat.
In the past six months, AbbVie has shown the strongest performance with a 9.4% increase, while Novo Nordisk has declined the most at 36.3%.