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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. 

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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

WEEKLY NEWS RECAP #Phispers

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BioMarin inks US$ 4.8 billion rare disease deal with Amicus; FDA approves pill version of Novo’s Wegovy
PharmaCompass wishes its readers a very Happy New Year and brings news from the last two weeks in this double-dose edition of Phispers.US President Donald Trump announced agreements with major drugmakers to lower medicine prices for the Medicaid program and cash-pay patients, with discounts tied to the lowest levels paid by other developed nations.On December 19, BioMarin agreed to buy rare disease specialist Amicus Therapeutics for US$ 4.8 billion, adding Fabry and Pompe disease drugs to its portfolio. AstraZeneca signed a deal worth up to US$ 2 billion with China-based Jacobio Pharma for an early-stage pan-KRAS cancer drug. Japan’s Shionogi moved to strengthen its rare disease focus with a US$ 2.5 billion acquisition of Tanabe Pharma’s Radicava ALS business. And Sanofi announced the acquisition of US biotech Dynavax Technologies for around US$ 2.2 billion (€ 1.9 billion).On the regulatory front, the US Food and Drug Administration (FDA) approved a pill version of Novo Nordisk’s Wegovy for weight management, cleared Cytokinetics’ Myqorzo for a rare obstructive heart condition and approved Omeros’ Yartemlea to treat a transplant-associated complication. It also gave a nod to Vanda’s drug for motion sickness in more than 40 years, and expanded Agios’ drug as the first oral treatment for anemia. And the agency placed a partial clinical hold on Merck and Daiichi Sankyo’s late-stage lung cancer trial due to safety concerns. Separately, Ultragenyx’s drug missed a key trial goal in rare bone disorder.Trump, drugmakers strike deals to cut medicine prices for Medicaid programUS President Donald Trump and nine major drugmakers have announced agreements to lower prescription drug prices for the government’s Medicaid program and for cash payers. These prices will be in line with the lowest prices paid by other developed nations (known as ‘most-favored-nation’ pricing).Companies that signed the deals with the Trump administration include Bristol Myers Squibb, Gilead Sciences, Merck, Genentech, Novartis, Amgen, Boehringer Ingelheim, Sanofi, and GSK. These companies pledged to invest over US$ 150 billion in the US. However, it was not clear if this included their earlier commitments.Under the agreements, discounts could reach up to 70 percent of the list prices, and will be offered potentially through the TrumpRx website, expected to be launched this year.BioMarin to buy Amicus Therapeutics for US$ 4.8 bn in rare disease dealBioMarin has agreed to acquire Amicus Therapeutics in an all-cash transaction valued at approximately US$ 4.8 billion. This is the largest deal signed by BioMarin in its 28-year history.The deal adds two approved and fast-growing rare disease medicines to BioMarin’s portfolio: Galafold (migalastat) for Fabry disease and, Pombiliti-Opfolda (cipaglucosidase alfa-atga + miglustat), a combination treatment for Pompe disease. BioMarin will also gain US rights to DMX-200, an experimental kidney disease drug.Fabry disease is caused by a lack of an enzyme that breaks down fats, causing fat buildup in blood vessels and tissues. Pompe disease involves a buildup of glycogen in cells, leading to muscle weakness and other symptoms.Astra inks oncology deal with China’s Jacobio: AstraZeneca has signed a deal with China-based Jacobio Pharma to develop an early-stage cancer drug that targets multiple KRAS mutations. Under the agreement, AstraZeneca will pay US$ 100 million upfront and could provide up to about US$ 2 billion more in milestone payments, along with royalties.Shionogi to buy Tanabe’s ALS business for US$ 2.5 bn: Japanese drugmaker Shionogi has agreed to acquire Tanabe Pharma’s ALS (amyotrophic lateral sclerosis) business for US$ 2.5 billion, including both the oral formulation of Radicava (edaravone) and its intravenous version. As part of the deal, Tanabe Pharma will set up a new company to hold the rights to Radicava (an FDA-approved ALS treatment), which Shionogi will then acquire outright.Sanofi to buy Dynavax: Sanofi has announced the acquisition of US biotech Dynavax Technologies for around US$ 2.2 billion (€ 1.9 billion). The deal will add an adult hepatitis B vaccine and a promising experimental shingles shot to Sanofi’s portfolio.FDA okays pill version of Novo’s Wegovy, Cytokinetics med for rare heart conditionNovo Nordisk has got a leg up against its competitors in the fast-growing obesity treatment market with the FDA approving the pill version of Wegovy (semaglutide) for weight management and reduction of cardiovascular risk in overweight/obese patients. The drug can be given at a maintenance dose of up to 25 mg. Approves Cytokinetics’ Myqorzo for rare obstructive heart condition: FDA has approved Cytokinetics’ oral drug Myqorzo (aficamten) to treat obstructive hypertrophic cardiomyopathy, a rare heart condition in which the heart muscle becomes abnormally thick, making it harder for the heart to pump blood. The disease can cause symptoms such as shortness of breath, chest pain and dizziness. Myqorzo is Cytokinetics’ first FDA-approved product and only the second drug in its class to be approved in the US. The FDA approval includes a boxed warning about the risk of heart failure.Okays Omeros’ drug to treat transplant associated complication: FDA has approved Omeros’ drug — Yartemlea  (narsoplimab-wuug) — for a dangerous transplant complication, known as transplant-associated thrombotic microangiopathy (TA-TMA). This serious complication develops after stem cell transplants.Clears Agios’ Aqvesme as first oral treatment for anemia: The FDA approved an expanded use of Agios Pharmaceuticals’ drug mitapivat, sold under the brand name Aqvesme, to treat anemia in adults with alpha- or beta-thalassemia. The approval makes Aqvesme the first oral treatment for anemia in both transfusion-dependent and non-transfusion-dependent thalassemia patients.Gives nod to Vanda’s drug for motion-induced vomiting: Vanda Pharmaceuticals said the FDA has approved its drug Nereus (tradipitant) for the prevention of motion-induced vomiting, marking the first FDA approval for this condition in more than 40 years. Existing options for motion sickness include patches and over-the-counter medicines, but no new treatment had been approved in decades.Partial hold on Merck-Daiichi’s lung cancer trial: FDA has placed a partial clinical hold on a late-stage trial of ifinatamab deruxtecan (I-DXd), an experimental cancer drug being developed by Merck and Daiichi Sankyo. The decision follows reports of a higher-than-expected number of patient deaths linked to a serious lung condition.Ultragenyx’s drug misses key trial goal in rare bone disorderUltragenyx Pharmaceutical said late-stage trials of its experimental drug setrusumab failed to meet their main goal, causing the company’s shares to plunge more than 43 percent to a record low on the day. The drug was being tested in children and young adults with osteogenesis imperfecta, a rare genetic bone disease that makes bones fragile and prone to fractures. Ultragenyx ran the studies with partner Mereo BioPharma.  

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https://www.pharmacompass.com/radio-compass-phisper/biomarin-inks-us-4-8-billion-rare-disease-deal-with-amicus-fda-approves-pill-version-of-novo-s-wegovy

#Phispers by PHARMACOMPASS
01 Jan 2026

NEWS #PharmaBuzz

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https://www.businesswire.com/news/home/20260522186975/en/Avanzanite-Biosciences-Partner-Agios-Announces-PYRUKYND-mitapivat-Approval-in-the-European-Union-for-Adults-with-Thalassaemia

BUSINESSWIRE
22 May 2026

https://www.globenewswire.com/news-release/2026/05/22/3300143/31990/en/agios-pyrukynd-mitapivat-approved-in-the-european-union-for-adults-with-thalassemia.html

GLOBENEWSWIRE
22 May 2026

https://www.globenewswire.com/news-release/2026/05/12/3292999/31990/en/agios-to-present-new-data-at-eha-2026-reinforcing-the-significant-therapeutic-impact-of-mitapivat-across-multiple-rare-hemolytic-anemias.html

GLOBENEWSWIRE
12 May 2026

https://www.globenewswire.com/news-release/2026/05/12/3293011/31990/en/agios-submits-snda-to-fda-for-u-s-accelerated-approval-of-mitapivat-in-sickle-cell-disease.html

GLOBENEWSWIRE
12 May 2026

https://www.globenewswire.com/news-release/2026/04/29/3283559/31990/en/agios-reports-first-quarter-2026-financial-results-and-provides-business-update.html

GLOBENEWSWIRE
29 Apr 2026

https://www.globenewswire.com/news-release/2026/03/31/3265325/31990/en/Agios-Advances-Mitapivat-Toward-Potential-U-S-Accelerated-Approval-in-Sickle-Cell-Disease-Following-Pre-sNDA-Meeting-with-FDA.html

GLOBENEWSWIRE
31 Mar 2026