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DATA COMPILATION #PharmaFlow

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FDA approvals rise 49% in 2023; CRISPR’s gene editing therapy sees light of day
In 2022, when the US Food and Drug Administration (FDA) was reeling under the impact of the pandemic, new drug approvals by the agency dropped by 26 percent. But last year, FDA’s new drug approvals rebounded by an impressive 49 percent, with the Center for Drug Evaluation and Research (CDER) approving 55 new drugs in 2023. Of them, 36 percent were considered first-in-class, while small molecules made up for 62 percent of the total drugs approved (i.e. 34). FDA’s Center for Biologics Evaluation and Research (CBER) okayed 19 biologics in 2023 compared to eight in the previous year.The first half of 2023 saw the debut of vaccines for the all-too-common respiratory syncytial virus (RSV). Among the other notable approvals in H1 was Biogen and Eisai’s Alzheimer’s drug Leqembi (lecanemab). Out of the total 55 drug approvals, 29 came in H2 2023. This includes Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy that relies on the Nobel Prize-winning CRISPR gene-editing technology. Casgevy has been approved as a treatment for sickle-cell disease (SCD) and β-thalassemia.While FDA witnessed a sharp rise in approvals in 2023, many other drug regulators didn’t. The European Medicines Agency (EMA) granted marketing authorization to 32 novel drugs in 2023, a fall from 33 in 2022. Similarly, Health Canada’s approvals in 2023 decreased to 38, compared to 45 in the previous year.As usual, oncology topped the list of drug approvals by therapeutic area, at 39 (as opposed to 35 in 2022). Rare diseases was the second most popular therapeutic area for drug approvals. With drugmakers clearly paying heed to the unmet needs of patients suffering from rare diseases, this therapeutic area sprinted from a 9 percent share and the fourth position among new approvals in 2022 to an impressive 34 percent share in 2023. A quarter of the new drug approvals were in infectious diseases, followed by immunology (19 percent) and neurology (7 percent).View New Drug Approvals in 2023 with Estimated Sales (Free Excel Available) Casgevy, postpartum depression drug Zurzuvae emerge as potential blockbustersGene therapy Casgevy, postpartum depression (PPD) med Zurzuvae, blood cancer med Elrexfio and ulcerative colitis drug Velsipity were some of the prominent approvals of 2023.Britain’s Medicines and Healthcare products Regulatory Agency was the first to okay Casgevy in November as a cure for SCD and β-thalassemia. Soon, the FDA approved it for SCD. In January this year, the American agency also approved it for transfusion-dependent β-thalassemia (TDT). Analysts estimate Casgevy to generate US$ 2.6 billion in peak sales, says Nature. Biogen and Sage’s PPD therapy Zurzuvae became the first and only FDA-approved pill for the condition that can be life-threatening for both the mother and the child. Global sales of Zurzuvae are forecast to hit US$ 1.28 billion by 2028.In August, Pfizer’s Elrexfio (elranatamab) became the first “off-the-shelf” (ready-to-use) therapy in the US for multiple myeloma. The drug provides an option for patients with hard to treat or relapsed blood cancer and is estimated to bring in US$ 861 million in peak sales by 2028, says Nature.Pfizer also bagged another significant approval in October — its drug Velsipity (etrasimod) was greenlit by the FDA to treat adults with ulcerative colitis, an inflammatory bowel disease. Peak revenue for Velsipity is expected to come in at US$ 825 million, as per Evaluate.View New Drug Approvals in 2023 with Estimated Sales (Free Excel Available) Astra’s Truqap, GSK’s Ojjaara among top cancer therapies given FDA nod in H2In November, FDA approved AstraZeneca’s Truqap (capivasertib) in combination with the Anglo-Swedish drugmaker’s Faslodex (fulvestrant) for treating adult patients with hormone receptor (HR)-positive, HER2-negative locally advanced or metastatic breast cancer with one or more biomarker alterations. Evaluate Pharma forecasts peak Truqap sales to come in at about US$ 690 million.In September, FDA approved GSK’s Ojjaara (momelotinib) as the first and only treatment for myelofibrosis with anemia. Nearly all myelofibrosis patients are estimated to develop anemia over the course of the disease. Ojjaara is taken orally once a day.Other notable oncology treatments okayed by FDA in H2 2023 include Daiichi’s Vanflyta (quizartinib) in July to treat an aggressive blood cancer known as acute myeloid leukemia (AML). In August, FDA approved Janssen’s bispecific antibody Talvey (talquetamab-tgvs) for difficult-to-treat blood cancer. The agency approved two cancer therapies in November — BMS’ Augtyro (repotrectinib) for ROS1-positive non-small cell lung cancer (NSCLC) and Takeda’s targeted oral therapy Fruzaqla (fruquintinib) for adult patients with metastatic colorectal cancer (mCRC).View New Drug Approvals in 2023 with Estimated Sales (Free Excel Available) Rare disease drugs Santhera-Catalyst’s Agamree, Novo’s Rivfloza bag approval in H2In October, FDA approved Santhera Pharmaceuticals and Catalyst Pharma’s Agamree (vamorolone), an oral suspension treatment for Duchenne muscular dystrophy (DMD) in patients two years of age and older. This makes it the first drug fully approved in both the US and Europe for the muscle degeneration disorder. Agamree acts in a manner similar to other steroids, which are the standard of care for the inherited rare disease. However, it causes fewer side effects.FDA also okayed Novo Nordisk’s once-a-month injection Rivfloza (nedosiran) in October to treat a rare genetic condition — primary hyperoxaluria type 1 (PH1) — that causes recurring kidney stones.In November, the agency approved Takeda’s Adzynma (ADAMTS13, recombinant-krhn) as the first treatment for both adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP), a rare genetic blood disorder. Other noteworthy FDA approvals in H2 2023 for rare blood diseases include Novartis’ Fabhalta and bluebird bio's Lyfgenia. Fabhalta is the first oral monotherapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria, a rare disease that causes symptoms such as hemolytic anemia, hemoglobinuria (excretion of hemoglobin in the urine), fatigue, shortness of breath etc. Lyfgenia is the first cell-based gene therapy for the treatment of SCD in patients 12 years and older. Similarly, another rare disease drug — Regeneron’s Veopoz —  bagged FDA approval in August last. Veopoz treats CHAPLE disease, an ultra-rare disease in which patients have severe gastrointestinal problems.View New Drug Approvals in 2023 with Estimated Sales (Free Excel Available) Our viewAfter a lull in 2022, new drug approvals have finally gathered momentum. The good news is that this year, several pathbreaking drugs are coming up for approval, such as Madrigal Pharmaceuticals’ resmetirom (the first treatment for NASH with liver fibrosis), Merck’s sotatercept (a treatment for pulmonary arterial hypertension), Lilly’s donanemab for Alzheimer’s disease and Karuna Therapeutics’ drug to treat schizophrenia. Let’s hope 2024 turns out to be an even bigger year for new drug approvals.

Impressions: 3844

https://www.pharmacompass.com/radio-compass-blog/fda-approvals-rise-49-in-2023-crispr-s-gene-editing-therapy-sees-light-of-day

#PharmaFlow by PHARMACOMPASS
01 Feb 2024

STOCK RECAP #PipelineProspector

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Pipeline Prospector April 2025: Merck KGaA buys SpringWorks for US$ 3.9 bn; Swiss giants lead pharma migration to US soil
April witnessed considerable volatility in pharmaceutical stocks as the US President Donald Trump’s administration signaled major shifts in drug pricing policies and import regulations. These include Trump’s proposed international reference pricing to align US drug prices with lower costs in other developed countries, a national security investigation into pharmaceutical imports that could lead to tariffs of 25 percent or more on foreign-made drugs and, a sweeping executive order to reshape Medicare drug pricing. However, as the month drew to a close, key pharmaceutical indices rebounded and were back in the black. The Nasdaq Biotechnology Index (NBI) rose 0.44 percent from 4,241.91 to 4,260.42. The SPDR S&P Biotech ETF (XBI) rose 2.58 percent — from 80.86 to 82.95. And the S&P Biotechnology Select Industry Index (SPSIBI) increased 0.91 percent — from 6,319.47 to 6,376.95. However, stocks of most drugmakers ended the month in the red.  Access the Pipeline Prospector Dashboard for April 2025 Newsmakers (Free Excel) Trump’s tariff threats prompt drug majors to announce investments in US The unrest created by Trump’s tariff threat on drug imports was palpable across the world. In Europe, CEOs of nearly three dozen global drugmakers, including Pfizer, Eli Lilly, and AstraZeneca, warned European Commission President Ursula von der Leyen that without "rapid, radical policy change", over US$ 100 billion in pharma R&D and manufacturing could leave the European Union. These drugmakers are pushing for abandonment of price controls to match US pricing levels.  Global players are also rapidly reorienting their manufacturing strategies towards the US soil in order to avoid imminent tariffs on imported medicines. For instance, Swiss pharmaceutical giant Roche has announced a massive US$ 50 billion investment in the US over the next five years. Another Swiss drugmaker, Novartis, has committed US$ 23 billion to expand its US operations over the next five years. Meanwhile, AstraZeneca has said it will be shifting the production of some drugs sold in America from Europe to the US in order to avoid Trump’s tariffs. Even American drugmaker AbbVie plans to invest US$ 10 billion in US manufacturing over the next decade to avoid tariffs. Regeneron Pharmaceuticals has taken a different approach by announcing an investment exceeding US$ 3 billion through a 10-year strategic partnership with Fujifilm Diosynth Biotechnologies. This collaboration aims to nearly double Regeneron’s US production capacity for biologic medicines.  Access the Pipeline Prospector Dashboard for April 2025 Newsmakers (Free Excel)  Merck buys SpringWorks for US$ 3.9 bn; GSK inks US$ 2.7 bn deal with ABL Bio This was going to be a year of mergers and acquisitions. But with the imminent policy changes in America’s healthcare sector, M&A activity appears to have slowed down. However, the last days of April saw two M&A announcements — Merck KGaA entered into a deal to buy US biotech company SpringWorks Therapeutics for US$ 3.9 billion, and Novartis announced the acquisition of Regulus Therapeutics for up to US$ 1.7 billion. The month saw several licensing deals. GSK signed a licensing agreement valued at approximately £ 2.08 billion (US$ 2.66 billion) with South Korea’s ABL Bio for exclusive access to ABL’s innovative Grabody-B brain delivery platform, a technology designed to enhance the penetration of therapeutic antibodies across the notoriously selective blood-brain barrier (BBB). The BBB acts as the body’s natural defense to protect the brain from toxins and pathogens. Similarly, Eli Lilly inked a US$ 1.4 billion licensing agreement with Sangamo Therapeutics to gain access to Sangamo’s proprietary neurotropic adeno-associated virus (AAV) capsid, STAC-BBB, which has shown remarkable potential in crossing the BBB. In the autoimmune and immunology space, Sanofi signed an exclusive licensing agreement with US biotech Earendil Labs valued at up to US$ 1.8 billion. The agreement centers on two next-generation bispecific antibodies, HXN-1002 and HXN-1003, discovered through Earendil's AI-driven high-throughput protein modeling platform.  Access the Pipeline Prospector Dashboard for April 2025 Newsmakers (Free Excel)  Lilly’s obesity pill matches Ozempic in trial; Novo seeks FDA nod for semaglutide pill In the market for obesity meds, Eli Lilly’s experimental weight-loss pill, orforglipron, demonstrated efficacy that was comparable to its rival Novo Nordisk’s injectable Ozempic. Analysts view orforglipron pill as a potential game-changer in obesity treatment. Meanwhile, Novo Nordisk has sought FDA approval for a 25-mg pill version of its weight loss medication semaglutide. And Rhythm Pharmaceuticals’ drug, Imcivree (setmelanotide), achieved primary endpoint in a late-stage trial for acquired hypothalamic obesity, a rare condition resulting from damage to the brain’s hypothalamus. Meanwhile, in a high profile trial failure, Pfizer discontinued development of its experimental oral weight-loss pill, danuglipron, following a case of potential drug-induced liver injury in a clinical trial participant. Another significant disappointment came from BMS’ investigational schizophrenia drug, Cobenfy (xanomeline and trospium chloride) — it failed to meet the primary endpoint in a pivotal late-stage trial evaluating its use as an adjunctive treatment for patients who no longer respond to standard antipsychotics. Adding to the month’s disappointments, FDA declined to approve Aldeyra Therapeutics’ dry eye disease treatment, reproxalap, for the second time.  Access the Pipeline Prospector Dashboard for April 2025 Newsmakers (Free Excel)  Amgen’s Uplizna, Sanofi’s Dupixent among noteworthy FDA approvals Amgen received a groundbreaking approval for Uplizna (inebilizumab-cdon) as the first and only treatment for immunoglobulin G4-related disease (IgG4-RD), a rare and chronic immune-mediated inflammatory condition. Analysts estimate that Uplizna could generate global annual sales of US$ 1.3 billion by 2030 for the IgG4-RD indication alone.  Sanofi and Regeneron received FDA approval for Dupixent (dupilumab) to treat chronic spontaneous urticaria (CSU) in patients aged 12 and older who remain symptomatic despite antihistamine therapy. Sanofi’s bestselling drug is now FDA-approved for seven indications. As the month drew to a close, FDA approved Abeona Therapeutics’ gene therapy — Zevaskyn (prademagene zamikeracel) — for a rare skin disorder known as recessive dystrophic epidermolysis bullosa. It also approved Johnson & Johnson’s Imaavy (nipocalimab-aahu), a drug that treats patients aged 12 years and older with a form of generalized myasthenia gravis (gMG) — an immunity-related disorder that weakens the skeletal muscles, especially those in the eyes, mouth, throat and limbs.  Access the Pipeline Prospector Dashboard for April 2025 Newsmakers (Free Excel)  Our view The US economy contracted by 0.3 percent during the first quarter of 2025, as businesses panicked and imported goods to avoid higher costs. The US dollar has been weakening, and has hit a fresh low against the euro, Japanese yen and the Swiss franc. This will reduce the profit of foreign companies from their US divisions once they remit their earnings back to their home country. A study undertaken by Ernst & Young has predicted that a 25 percent US tariff on drug imports would increase drug cost by nearly US$ 51 billion annually, thereby boosting drug prices in America by 12.9 percent. These and other cues only point to the fact that Trump administration’s policies will hurt the US. As of today, a rollback or a softening of stand looks like a distant possibility. Access the Pipeline Prospector Dashboard for April 2025 Newsmakers (Free Excel)  

Impressions: 4140

https://www.pharmacompass.com/pipeline-prospector-blog/pipeline-prospector-april-2025-merck-kgaa-buys-springwork-for-us-3-9-bn-swiss-giants-lead-pharma-migration-to-us-soil

#PharmaFlow by PHARMACOMPASS
01 May 2025

NEWS #PharmaBuzz

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https://endpts.com/fda-approves-amgens-uplizna-in-igg4-related-disease/

ENDPOINTS
04 Apr 2025

https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=overview.process&ApplNo=761142

FDA
03 Apr 2025

https://www.prnewswire.com/news-releases/amgen-presents-positive-phase-3-data-for-uplizna-inebilizumab-cdon-in-generalized-myasthenia-gravis-gmg-at-aanem-2024-302275645.html

PR NEWSWIRE
15 Oct 2024

https://www.reuters.com/business/healthcare-pharmaceuticals/amgens-drug-meets-main-goal-late-stage-study-rare-disease-2024-06-05/

Sriparna Roy REUTERS
06 Jun 2024

https://www.prnewswire.com/news-releases/amgen-announces-positive-results-for-phase-3-registrational-trial-evaluating-uplizna-inebilizumab-cdon-for-treatment-of-immunoglobulin-g4-related-disease-igg4-rd-302164333.html

PR NEWSWIRE
05 Jun 2024

https://www.businesswire.com/news/home/20231002226886/en

BUSINESSWIRE
02 Oct 2023