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DATA COMPILATION #PharmaFlow

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Top first-in-class drug candidates of 2025: Ionis’ donidalorsen, Sanofi’s fitusiran, Cytokinetics’ aficamten await FDA approval
First‑in‑class drugs are therapies with entirely new approaches that improve patient outcomes and fundamentally change treatment paradigms.This week, PharmaCompass brings a compilation of 2025’s most promising first-in-class drug candidates. With their US Food and Drug Administration (FDA) action dates in 2025, many of these drugs, once approved, hold the potential of becoming blockbusters.Ionis’ donidalorsen, Sanofi’s fitusiran, Cytokinetics’ aficamten poised to transform patient careIonis’ donidalorsen is an exciting candidate designed to tackle hereditary angioedema (HAE), a rare condition marked by unpredictable and potentially life‑threatening swelling episodes.This novel antisense oligonucleotide works by “silencing” the messenger RNA that codes for prekallikrein — a protein that triggers inflammation leading to swelling. Instead of blocking a protein once it’s made, donidalorsen stops the cell from producing it in the first place. This precision approach could mean fewer infusions and reduced side effects compared to traditional treatments.Another promising candidate is Sanofi and Alnylam Pharmaceuticals’ fitusiran. It employs the technology of small interfering RNA (siRNA) to lower levels of antithrombin — a natural protein that, when in excess, can impair the blood’s ability to clot. By reducing antithrombin, fitusiran rebalances the blood’s clotting process in patients with hemophilia A and B.Similarly, Cytokinetics’ aficamten is designed to treat obstructive hypertrophic cardiomyopathy (oHCM), a condition in which the heart muscle becomes abnormally thick, obstructing blood flow. Aficamten works by inhibiting cardiac myosin, a protein that drives heart muscle contraction. This helps relieve symptoms like shortness of breath and chest pain. If approved, aficamten is expected to rake in annual sales of US$ 2.8 billion by 2030.UroGen, J&J’s candidates eye US$ 5 bn markets; AbbVie, Summit’s meds target lung cancerUroGen Pharma’s UGN‑102 is poised to redefine the treatment of non‑muscle‑invasive bladder cancer. This innovative formulation uses a reverse thermal gel technology that transforms from a liquid into a gel once inside the bladder. This change ensures that the chemotherapeutic agent, mitomycin, remains in contact with the bladder lining for a longer period, increasing its effectiveness at eradicating cancer cells and reducing the risk of recurrence.For patients, UGN‑102 could mean a non‑surgical, outpatient treatment option that is less invasive than traditional surgical methods. If approved, UGN-102 could address a US market of approximately 82,000 patients annually, translating into a market opportunity exceeding US$ 5 billion, according to company estimates.Johnson & Johnson’s nipocalimab is a new treatment aimed at helping people with autoimmune diseases — a group of conditions where the body’s defense system mistakenly attacks its own healthy cells. Nipocalimab works by blocking the neonatal Fc receptor (FcRn), thereby lowering the levels of pathogenic antibodies that drive many autoimmune disorders. J&J had acquired this FcRn blocker in 2020 as part of its US$ 6.5 billion takeover of Momenta Pharmaceuticals. J&J is trialing the drug in as many as 10 indications and expects peak annual sales in excess of US$ 5 billion.AbbVie’s antibody-drug conjugate (ADC), telisotuzumab vedotin, is designed as a “guided-missile” to deliver toxic drugs directly to cancer cells while sparing healthy tissues. Telisotuzumab vedotin targets c‑Met, a receptor that is over-expressed in certain cancers such as non‑small cell lung cancer (NSCLC). There are currently no approved anti-cancer therapies specifically for c-Met over-expressing NSCLC.Summit Therapeutics’ ivonescimab is a cutting‑edge bispecific antibody designed specifically for NSCLC. Unlike traditional therapies that target a single molecule, ivonescimab binds simultaneously to a tumor-specific antigen on cancer cells and to a receptor on T cells, effectively bringing these two together so the body’s own immune system can recognize and attack the tumor. This dual engagement strategy helps overcome common mechanism tumors.Insmed’s Brensocatib is an innovative, first‐in‐class oral inhibitor of dipeptidyl peptidase‑1 (DPP‑1) that targets a key step in the inflammatory cascade. DPP‑1 is responsible for activating neutrophil serine proteases — enzymes that, when overactive, can cause significant lung tissue damage in conditions such as non‑cystic fibrosis bronchiectasis. Brensocatib is expected to clock annual sales of US$ 2.8 billion by 2030.Gepotidacin, an investigational antibiotic developed by GSK, is poised to make a significant impact in the treatment of uncomplicated urinary tract infections (uUTIs) and urogenital gonorrhea. If approved, it would be the first oral antibiotic in a new class for uUTIs in over two decades.Arrowhead, Regenxbio, Ultragenyx address rare, genetic diseasesArrowhead Pharmaceuticals is developing an innovative gene-targeting therapy — plozasiran. This drug uses RNA interference (RNAi) to interrupt the gene responsible for producing apolipoprotein C‑III (APOC3), a protein that regulates blood triglyceride levels. For people with severe hypertriglyceridemia or familial chylomicronemia syndrome (FCS) — a severe and rare genetic disease for which there are currently no FDA‑approved treatments — high triglyceride levels can lead to pancreatitis and other serious cardiovascular issues. Regenxbio’s RGX‑121 is a one‑time gene therapy candidate for Hunter syndrome (MPS 2), a rare, inherited disorder that causes progressive neurological decline. The therapy uses a harmless virus to deliver a functional copy of the gene encoding iduronate‑2‑sulfatase (I2S) directly into the central nervous system. By restoring the missing enzyme, RGX‑121 aims to slow or even reverse the harmful buildup of substances in the brain that drive the disease’s progression.Another promising candidate is Ultragenyx Pharmaceuticals’ one-time intravenous gene therapy UX111 for Sanfilippo syndrome type A (MPS 3A). This devastating disorder, which leads to severe neurodegeneration in children, currently has no effective treatment. UX111 uses an adeno‑associated virus (AAV) to deliver a working copy of the SGSH gene, the deficiency of which is responsible for the disease. By enabling the body to produce the missing enzyme, UX111 holds the promise of slowing or even halting the progression of neurodegeneration.Our viewThis year has already seen several first-in-class approvals, such as Vertex’s suzetrigine (Journavx), a pioneering non‐opioid analgesic redefining pain management; SpringWorks’ mirdametinib (Gomekli), a breakthrough MEK inhibitor for a genetic disorder that often leads to the growth of benign tumors along nerves and; AstraZeneca and Daiichi’s ADC datopotamab deruxtecan (Datroway).Despite the volatile political environment in the US and its impact on federal agencies like the FDA, we are hopeful that many of the first-in-class drugs mentioned in this article will make a debut in 2025. 

Impressions: 6854

https://www.pharmacompass.com/radio-compass-blog/top-first-in-class-drug-candidates-of-2025-ionis-donidalorsen-sanofi-s-fitusiran-cytokinetics-aficamten-await-fda-approval

#Phispers by PHARMACOMPASS
27 Feb 2025

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: 2623

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.globenewswire.com/news-release/2026/01/27/3226375/35409/en/Cytokinetics-Announces-MYQORZO-aficamten-Now-Available-in-the-U-S-for-the-Treatment-of-Adults-with-Symptomatic-Obstructive-Hypertrophic-Cardiomyopathy-to-Improve-Functional-Capacit.html

GLOBENEWSWIRE
27 Jan 2026
Myqorzo And Redemplo Approved In China
Myqorzo And Redemplo Approved In China

17 Jan 2026

// PRESS RELEASE

https://www.sanofi.com/en/media-room/press-releases/2026/2026-01-15-06-00-00-3219227

PRESS RELEASE
17 Jan 2026

https://www.prnewswire.com/news-releases/orsini-selected-as-specialty-pharmacy-partner-for-cytokineticss-myqorzo-aficamten-302659745.html

PR NEWSWIRE
13 Jan 2026

https://www.globenewswire.com/news-release/2026/01/12/3217380/0/en/CareMed-Has-Been-Selected-as-a-National-Specialty-Pharmacy-Provider-for-MYQORZO-aficamten.html

GLOBENEWSWIRE
12 Jan 2026

https://ir.cytokinetics.com/press-releases/press-release-details/2025/Cytokinetics-Announces-FDA-Approval-of-MYQORZO-aficamten-for-the-Treatment-of-Adults-with-Symptomatic-Obstructive-Hypertrophic-Cardiomyopathy-to-Improve-Functional-Capacity-and-Symptoms/default.aspx

PRESS RELEASE
19 Dec 2025

https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=219083

FDA
19 Dec 2025