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DATA COMPILATION #PharmaFlow

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FDA approvals drop 8% in 2025, with fewer blockbusters; Brinsupri, Rhapsido make it to first-in-class list
Our update for new drug approvals by the US Food and Drug Administration (FDA) in the first half (H1) of 2025 had pointed out how upheavals at the agency had impacted its functioning, with drug approvals dropping by 24 percent.While the turbulence didn’t subside, approvals picked up considerably in the second half (H2) of 2025. FDA’s Center for Drug Evaluation and Research (CDER) approved 30 new drugs in H2 (as against 16 in H1), taking the total number of approvals for 2025 up to 46. This is an 8 percent drop compared with 50 approvals CDER had granted in 2024.During the year, CDER saw four new chiefs come and go — Jacqueline Corrigan-Curay (Jan-July 2025), George Tidmarsh (July-Nov 2025), Richard Pazdur (Nov-December 2025) and Tracy Beth Høeg (December 2025-present). In addition, the agency endured thousands of retrenchments. The US Department of Health and Human Services (HHS), under Health Secretary Robert F. Kennedy Jr., enforced funding cuts and changes in approval processes, especially for vaccines.Overall, we have been witnessing a steady drop in drug approvals by CDER — it approved 55 new drugs in 2023, 50 in 2024 and, 46 in 2025.Of the 46 new approvals in 2025, 20 were classified as first-in-class (therapies that use a new and unique mechanism of action), out of which 13 were approved in H2 2025. Overall, CDER approved 32 chemical entities and 14 biologics in 2025.In comparison, approvals by Health Canada and authorizations by the European Medicines Agency (EMA) rose in 2025. The EMA authorized 69 new therapies, up from 64 in 2024. Health Canada approved 46 new therapies in 2025, as compared with 28 in 2024.FDA’s Center for Biologics Evaluation and Research (CBER) approved 21 biologics in 2025, compared with 13 in 2024, of which eight were notable new drugs.Interestingly, both CDER and CBER rejected more drugs last year — 43 applications were rejected, including those for new drugs, supplemental filings, generics and biosimilars, as opposed to 29 in 2024. View New Drug Approvals in 2025 with Estimated Sales (Free Excel Available)Blockbuster drug approvals drop by 60%, European drugmakers score more FDA nods It wasn’t a year of blockbuster drug approvals — the agency approved a lower number of drugs that are expected to deliver sales of at least US$ 1 billion by 2030. While there were nearly 20 such drugs approved in 2024, the number dropped to just eight in 2025.Interestingly, European companies won many more approvals for new products than their counterparts in the US. GSK and Novartis achieved three approvals each, while Merck, Boehringer Ingelheim and Bayer AG won two approvals each.The first-in-class drugs approved in H2 are: Insmed's Brinsupri (brensocatib), Ionis's Dawnzera (donidalorsen), Stealth Bio's Forzinity (elamipretide), Boehringer's Jascayd (nerandomilast), UCB's Kygevvi (doxecitine and doxribtimine), Bayer's Lynkuet (elinzanetant), Chimerix's Modeyso (dordaviprone), Innoviva's Nuzolvence (zoliflodacin), Arrowhead's Redemplo (plozasiran), Novartis' Rhapsido (remibrutinib), Otsuka's Voyxact (sibeprenlimab-szsi), Sanofi's Wayrilz (rilzabrutinib) and Omeros' Yartemlea (narsoplimab-wuug). View New Drug Approvals in 2025 with Estimated Sales (Free Excel Available)Insmed’s Brinsupri approved for chronic lung disease; Novartis’ Rhapsido okayed for urticariaAs has been the trend, the field of oncology saw the maximum drug approvals (15), followed by rare diseases and disorders (7) and immunology (4). Three categories — infections and infectious diseases, respiratory diseases and cardiology/vascular diseases — saw three drug approvals each.Among the first-in-class approvals was Insmed’s Brinsupri (brensocatib), the first treatment for non-cystic fibrosis bronchiectasis, a chronic lung disease. Nature has estimated peak sales of US$ 6.3 billion for this treatment. Brinsupri was approved by the EMA in November.Another notable approval was granted to Novartis’ Rhapsido (remibrutinib), approved to treat the skin condition chronic spontaneous urticaria in adults who remain symptomatic despite treatment with antihistamines. Rhapsido is the first oral alternative to injectable treatments and has 2030 sales forecasts of US$ 2.1 billion. Novartis is also testing the drug for other immune conditions.A new drug that is estimated to bring in the maximum sales is Merck’s Keytruda Qlex, a subcutaneous formulation of pembrolizumab plus berahyaluronidase alfa approved for various solid tumors. While top-selling cancer drug Keytruda (pembrolizumab) was first approved in 2014, this is the first approval for an engineered variant of the hyaluronidase enzyme (berahyaluronidase alfa). Therefore, this combination counts as a novel approval. View New Drug Approvals in 2025 with Estimated Sales (Free Excel Available)Fondazion’s CGT okayed for Wiskott-Aldrich syndrome; three drugs approved for hereditary angioedema After a record nine new cell and gene therapy (CGT) approvals in 2024, only five CGTs were approved in 2025 (the lowest since 2022). With the exception of Novartis’ Itvisma (onasemnogene abeparvovec-brve), all other CGT approvals went to relatively lesser known companies such as Abeona, Precigen, Neurotech and Fondazione Telethon (an Italian firm that became the first nonprofit to get a CGT approval from the FDA).Fondazione Telethon’s Waskyra (etuvetidigene autotemcel) became the first gene therapy for Wiskott-Aldrich syndrome (WAS), a rare immune disorder that causes frequent infections, bleeding issues, and eczema. The one-time treatment uses patients’ own blood stem cells that are modified to restore the faulty WAS gene. Abeona’s Zevaskyntm (prademagene zamikeracel) became the first and only autologous cell sheet-based gene therapy indicated for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (a rare, genetic skin disorder).Patients with hereditary angioedema (or HAE, a genetic disorder characterized by recurrent episodes of severe swelling) gained access to three new drugs, including two first-in-class drugs — CSL Behring’s Andembry (garadacimab) and Ionis Pharma’s Dawnzera (donidalorsen). The third drug approved to treat HAE is KalVista Pharma’s Ekterly (sebetralstat). View New Drug Approvals in 2025 with Estimated Sales (Free Excel Available)Our viewThe FDA has been bringing about considerable changes to its processes. In June, it launched the Commissioner’s National Priority Vouchers (CNPV) program to cut review timelines from the usual 10 to 12 months to two months. But the recent rejection of Disc Medicine’s application for bitopertin as a treatment for a rare blood disorder under the program took four months to materialize. Not only did the rejection come in late, it was based on information already known to the FDA at the time of granting the coveted voucher, raising controversy around the program.The agency is going to bring about more changes soon. It has proposed a plausible mechanism pathway for rare and ultra-rare diseases where randomized controlled trials are not feasible. And in April, it announced plans to phase out animal toxicity testing in the development of monoclonal antibody therapies and other drugs. The agency is increasingly relying on AI, and recently  announced the deployment of agentic AI capabilities for all employees. It will be interesting to see how these changes impact drug approvals in 2026.

Impressions: 1747

https://www.pharmacompass.com/radio-compass-blog/fda-approvals-drop-8-in-2025-with-fewer-blockbusters-brinsupri-rhapsido-make-it-to-first-in-class-list

#PharmaFlow by PHARMACOMPASS
26 Feb 2026

STOCK RECAP #PipelineProspector

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Pipeline Prospector 2025 highlights: FDA approves pill version of Novo’s Wegovy; BioMarin acquires Amicus for US$ 4.8 bn
Even though the biotech indices delivered strong gains through 2025, December closed on a muted note. The Nasdaq Biotechnology Index (NBI) declined 1.12 percent in December, slipping from 5,772.06 to 5,707.20. The SPDR S&P Biotech ETF (XBI) was largely flat, edging up 0.07 percent from 121.85 to 121.93. Meanwhile, the S&P Biotechnology Select Industry Index (SPSIBI) fell 0.62 percent, from 9,588.09 to 9,528.24.Overall, biotech indices showed sharp gains during 2025. From January to December, NBI rose 31.48 percent, climbing from 4,340.87 to 5,707.20. XBI gained 34.06 percent over the same period, increasing from 90.95 to 121.93, while SPSIBI jumped 36.52 percent, advancing from 7,023.04 to 9,528.24.The year saw the world’s largest pharmaceutical market put pressure on drugmakers to invest and reduce drug prices. In December, US President Donald Trump and nine major drugmakers — including Bristol Myers Squibb, Gilead Sciences, Merck, Genentech, Novartis, Amgen, Boehringer Ingelheim, Sanofi and GSK — announced agreements to lower prescription drug prices for the Medicaid program and for cash-paying patients under a most-favored-nation (MFN) pricing framework. These announcements built on earlier MFN pricing agreements the administration had signed with Pfizer and AstraZeneca.During 2025, several big drugmakers announced new US investments to avert the looming threat of import tariffs. In all, drugmakers pledged over US$ 370 billion in the US in 2025.In December, the US also finalized a trade agreement with the UK, eliminating US tariffs on British pharmaceutical products and medical technology for at least three years in return for Britain spending more on medicines and overhauling how it values drugs. Access the Pipeline Prospector Dashboard for December 2025 Newsmakers (Free Excel) FDA approves pill version of Novo’s Wegovy, GSK’s treatment for eosinophilic asthma okayedRegulatory approvals remained a major theme in December 2025. Novo Nordisk (stock up 4 percent in December) gained an edge in the obesity market after the FDA approved the oral pill version of its blockbuster Wegovy (semaglutide) for weight management and reduction of cardiovascular risk in overweight and obese patients. The drug is approved for maintenance doses of up to 25 mg. On December 16, GSK secured FDA approval for Exdensur (depemokimab) as an add-on maintenance treatment for patients aged 12 and older with eosinophilic asthma. During the same week, UK regulators approved Exdensur for both asthma and nasal polyps. GSK also received FDA approval for a label expansion of Blujepa (gepotidacin) — now approved as a treatment for gonorrhea for the same age bracket.California-based Innoviva received FDA approval for Nuzolvence (zoliflodacin), a first-in-class oral antibiotic for uncomplicated gonorrhea for patients aged 12 and older. Milestone Pharmaceuticals secured FDA approval for Cardamyst (etripamil), becoming the the first and only approved nasal spray to treat acute symptomatic episodes of paroxysmal supraventricular tachycardia (PSVT), a type of abnormal heart rhythm.Cytokinetics secured FDA approval for Myqorzo (aficamten), an oral treatment for obstructive hypertrophic cardiomyopathy. The approval marks the company’s first FDA-cleared product.Vanda Pharmaceuticals received FDA approval for Nereus (tradipitant) to prevent motion-induced vomiting, becoming the first new FDA-approved treatment for the condition in more than 40 years.US-based Agios Pharmaceuticals won FDA approval to expand the use of mitapivat, sold as Aqvesme, to treat anemia in adults with alpha- or beta-thalassemia, making it the first oral therapy for anemia in both transfusion-dependent and non-transfusion-dependent patients. Access the Pipeline Prospector Dashboard for December 2025 Newsmakers (Free Excel) BioMarin acquires Amicus for US$ 4.8 bn, Sanofi buys Dynavax, Shionogi bags Tanabe’s ALS bizDecember 2025 witnessed some mid-sized deals. BioMarin Pharmaceutical (stock up 6 percent) agreed to acquire Amicus Therapeutics in an all-cash transaction valued at approximately US$ 4.8 billion, marking the largest deal in BioMarin’s 28-year history. The acquisition adds two approved and fast-growing rare disease therapies — Galafold (migalastat) for Fabry disease and the Pompe disease combination treatment Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) — to BioMarin’s portfolio.Sanofi announced the acquisition of Dynavax Technologies for around US$ 2.2 billion (€1.9 billion). The transaction, all cash, strengthens Sanofi’s vaccine portfolio with an approved adult hepatitis B vaccine and an experimental shingles shot. In all, Sanofi cracked over a dozen deals in 2025.Japan-based Shionogi agreed to acquire Tanabe Pharma’s ALS business for US$ 2.5 billion, gaining both the oral and intravenous formulations of Radicava (edaravone), a US Food and Drug Administration (FDA)-approved treatment for amyotrophic lateral sclerosis (a progressive neurodegenerative disorder). Under the deal, Tanabe Pharma will set up a new company in the US to hold the rights to Radicava, which Shionogi will then acquire outright. Access the Pipeline Prospector Dashboard for December 2025 Newsmakers (Free Excel) Lilly to build new API plant in Alabama for its GLP-1 drug; Ultragenyx faces phase 3 setbackThe year 2025 was a good year for Eli Lilly — it inked nearly 30 deals through the year and became the first healthcare company in the world to reach a market capitalization of US$ 1 trillion in November. Lilly closed the year by pledging over  US$ 6 billion towards building a new API manufacturing facility in Huntsville, Alabama. The plant will produce small-molecule and peptide medicines, including orforglipron, Lilly’s oral GLP-1 weight-loss drug. Earlier in the year, Lilly had committed US$ 27 billion towards expanding US manufacturing capacity. After failing to bag an FDA approval for its rare disease gene therapy in July, Ultragenyx Pharmaceutical faced yet another setback in December when a late-stage trial of its experimental drug setrusumab failed to meet its primary endpoint in children and young adults with osteogenesis imperfecta (brittle bone disease). The trials were conducted in partnership with Mereo BioPharma. Ultragenyx’s stock was down 33 percent in December.There was some good news for patients suffering from Duchenne muscular dystrophy (DMD). Capricor Therapeutics reported positive phase 3 results for its DMD cell therapy — deramiocel. This success comes after Sarepta’s gene therapy Elevidys (delandistrogene moxeparvovec-rokl) suffered a setback in June, following patient deaths. Access the Pipeline Prospector Dashboard for December 2025 Newsmakers (Free Excel) Our viewThe pharmaceutical industry is growing at a healthy rate, and is expected to attain a size of US$ 1.19 trillion in 2026 (up from US$ 1.16 trillion in 2025). The industry is also expected to witness innovations, especially in fields such as weight management, rare diseases and oncology.However, we do expect some tug-of-war on drug prices. According to a Reuters report, drugmakers plan to raise US prices on at least 350 branded medications including some vaccines, even as the US administration pressures them for price cuts.On the geopolitical front, 2026 began with the US military capturing Venezuelan president Nicolás Maduro. Such developments can add to the volatility and uncertainties in the market, impacting supply chains, energy prices and investor sentiments. 

Impressions: 3868

https://www.pharmacompass.com/pipeline-prospector-blog/pipeline-prospector-2025-highlights-fda-approves-pill-version-of-novo-s-wegovy-biomarin-acquires-amicus-for-us-4-8-bn

#PharmaFlow by PHARMACOMPASS
08 Jan 2026

NEWS #PharmaBuzz

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https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=219491

FDA
12 Dec 2025

https://www.businesswire.com/news/home/20251212683585/en/U.S.-FDA-Approves-NUZOLVENCE-zoliflodacin-a-First-in-Class-Single-dose-Oral-Antibiotic-for-the-Treatment-of-Uncomplicated-Urogenital-Gonorrhea-in-Adults-and-Adolescents

BUSINESSWIRE
12 Dec 2025

https://www.pharmiweb.com/press-release/2025-10-20/innoviva-specialty-therapeutics-announces-oral-presentation-featuring-new-analyses-from-the-zoliflod

PHARMIWEB
20 Oct 2025

https://www.pharmiweb.com/press-release/2025-10-07/innoviva-specialty-therapeutics-to-deliver-six-presentations-from-its-infectious-diseases-and-critic

PHARMIWEB
07 Oct 2025

https://www.businesswire.com/news/home/20250612432209/en/FDA-Grants-Priority-Review-for-Zoliflodacin-New-Drug-Application-for-the-Treatment-of-Uncomplicated-Gonorrhea-and-Assigns-Target-PDUFA-Date-of-December-15-2025

BUSINESSWIRE
12 Jun 2025

https://www.businesswire.com/news/home/20250610102070/en/Innoviva-Specialty-Therapeutics-Receives-FDA-New-Drug-Application-Acceptance-for-Zoliflodacin-a-First-in-Class-Oral-Antibiotic-for-Uncomplicated-Gonorrhea-in-Adults

BUSINESSWIRE
10 Jun 2025