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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.

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STOCK RECAP #PipelineProspector

Pipeline Prospector Oct 2024: Lundbeck acquires Longboard for US$ 2.6 bn; molecular glue degrader tech witnesses dealmaking

In October, several pharma companies posted their third quarter (Q3) results. Drugmakers like Pfizer, BMS, Roche, Novartis, Sanofi, Merck and Incyte reported higher-than-expected Q3 earnings, beating analyst expectations. Despite these healthy results, pharma indices continued on their downward journey that had begun in September. The Nasdaq Biotechnology Index (NBI) dropped 2.6 percent from 4,771.85 to 4,650.07. The SPDR S&P Biotech ETF (XBI) fell 1.6 percent from 98.61 to 97.03, and the S&P Biotechnology Select Industry Index (SPSIBI) decreased 1.9 percent from 7,707.4 to 7,561.29. Access the Pipeline Prospector Dashboard for October 2024 Newsmakers (Free Excel) Lundbeck buys Longboard, AbbVie picks up Aliada, Merck acquires Modifi in US$ 1 bn+ deals In mergers and acquisitions, Denmark’s Lundbeck agreed to buy California-based Longboard Pharmaceuticals for US$ 2.6 billion. The acquisition centers around bexicaserin, a promising phase 3 candidate for rare epilepsies including Dravet syndrome and Lennox-Gastaut syndrome, with Lundbeck projecting potential peak sales of US$ 1.5 billion to US$ 2 billion for this asset. AbbVie acquired Boston-based Aliada Therapeutics for US$ 1.4 billion after reportedly outbidding at least three other drugmakers. The acquisition brings innovative blood-brain barrier technology to AbbVie’s portfolio, along with ALIA-1758, a phase 1 compound showing potential as a best-in-class therapy for Alzheimer’s disease. AbbVie also partnered with EvolveImmune Therapeutics in a potential US$ 1.4 billion deal (plus US$ 65 million upfront) to develop next-generation cancer biotherapeutics. The collaboration will leverage EvolveImmune’s innovative T-cell engager platform to create multispecific biologics targeting various oncology indications. Merck bolstered its oncology pipeline through the acquisition of Modifi Biosciences in a deal valued up to US$ 1.3 billion. The acquisition targets novel DNA modification therapeutics for challenging brain tumors, particularly glioblastomas. Merck also entered into a potential US$ 1.9 billion deal with Mestag Therapeutics to explore fibroblast therapies for inflammatory diseases, leveraging Mestag’s innovative platform. Access the Pipeline Prospector Dashboard for October 2024 Newsmakers (Free Excel) Pfizer, Novartis, Biogen sign molecular glue degrader deals; Astra in US$ 2 bn pact with CSPC There were at least three deals signed in October in the molecular glue degrader technology space. These degraders represent a novel therapeutic approach by facilitating the degradation of disease-causing proteins that are otherwise difficult to target with conventional drugs. First, Pfizer partnered Triana Biomedicines in a deal potentially exceeding US$ 1.5 billion, focusing on cancer applications. Second, Novartis committed US$ 150 million upfront to Monte Rosa Therapeutics in a deal worth up to US$ 2.25 billion. And Biogen tied up with Neomorph, pledging up to US$ 1.45 billion for neurological and immunological applications. Among other deals, AstraZeneca entered into a US$ 2 billion licensing agreement with CSPC Pharmaceutical Group for a novel lipid-lowering therapy, while Recordati acquired global rights to Sanofi’s Enjaymo for US$ 825 million upfront, with additional milestone payments of up to US$ 250 million. The Recordati-Sanofi deal focuses on cold agglutinin disease (CAD), a rare autoimmune disorder, and includes rights to sutimlimab, the first and only targeted therapy for CAD patients. Roche demonstrated its commitment to gene therapy advancement by expanding its collaboration with Dyno Therapeutics, committing over US$ 1 billion for adeno-associated virus (AAV) vector development, with an upfront payment of US$ 50 million. This expanded partnership builds on their initial 2020 collaboration and aims to accelerate the development of innovative gene therapies for neurological diseases. Access the Pipeline Prospector Dashboard for October 2024 Newsmakers (Free Excel) AbbVie wins FDA nod for subcutaneous Parkinson’s drug; Roche med okayed for breast cancer October marked significant advances in the treatment of several major diseases. FDA approved AbbVie’s Vyalev for advanced Parkinson’s disease. This innovative therapy, utilizing foscarbidopa and foslevodopa prodrugs, delivers round-the-clock symptom control through subcutaneous infusion. The treatment represents a major advancement in managing motor fluctuations in late-stage patients, with market analysts projecting peak sales exceeding US$ 2 billion. In the oncology space, a historic milestone was reached with FDA’s approval of Vyloy, the world’s first therapy targeting CLDN18.2 proteins in gastric cancer. Developed by Astellas, the drug was approved for use in combination with chemotherapy for treating advanced gastric or gastroesophageal junction adenocarcinoma in adults with HER2-negative, CLDN18.2-positive tumors. This approval validates Astellas’ strategic US$ 1.4 billion acquisition of Ganymed Pharmaceuticals in 2016. Roche strengthened its position in breast cancer treatment with the approval of Itovebi, an oral PI3K inhibitor for first-line treatment of advanced hormone receptor-positive, HER2-negative breast cancer with PIK3CA mutation. This approval positions Itovebi as a strong competitor to existing treatments like Novartis' Piqray and AstraZeneca's Truqap, with Roche projecting annual peak sales of US$ 2.3 billion. Novartis expanded the reach of its leukemia treatment Scemblix through an accelerated approval for newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase. This expansion significantly increases the eligible patient population by approximately four times, building on its existing approval as a third-line treatment. Pfizer expanded its presence in the respiratory syncytial virus (RSV) market as FDA broadened the approval of Abrysvo to include at risk adults aged 18 to 50 years, making it the first and only RSV vaccine authorized for this population. In hematology, Pfizer secured approval for Hympavzi marking its second hemophilia approval in six months. This approval follows the earlier authorization of Pfizer’s one-time gene therapy Beqvez for hemophilia B in April. Iterum Therapeutics received FDA approval for its new oral antibiotic, Orlynvah, designed to treat uncomplicated urinary tract infections (uUTIs) in adult women who have limited or no alternative oral antibacterial treatment options. This marks the first US approval for an oral penem antibiotic. Access the Pipeline Prospector Dashboard for October 2024 Newsmakers (Free Excel) Gilead withdraws Trodelvy, J&J discontinues late-stage study of its bladder cancer candidate In a notable development for bladder cancer treatment, Gilead Sciences announced the withdrawal of Trodelvy from the US market after the targeted therapy, which received accelerated FDA approval in 2021 for metastatic urothelial cancer, failed to demonstrate survival benefits in a crucial confirmatory study. Johnson & Johnson decided to discontinue the late-stage study of TAR-200, their investigational bladder cancer therapy, after interim analysis showed no superior benefits compared to standard chemo-radiation therapy. In neurology, Marinus Pharmaceuticals faced disappointment when their phase 3 trial of oral ganaxolone (Ztalmy) failed to meet its primary endpoint in reducing seizures associated with tuberous sclerosis complex (a genetic disorder). Marinus’ stock fell nearly 100 percent in October. Access the Pipeline Prospector Dashboard for October 2024 Newsmakers (Free Excel) Our view The last quarter was a good one for several drugmakers. Pfizer saw a surge in sales of its Covid drug Paxlovid to US$ 2.7 billion, encouraging it to up its guidance for Covid-related sales from US$ 8.5 billion to US$ 10.5 billion. For BMS, blockbusters like blood thinner Eliquis and cancer treatment Revlimid continued to bring in revenue. Merck’s growth was driven by the world’s top-selling drug, Keytruda, which saw revenue jump 17 percent compared to Q3 2023, beating analysts’ estimates. In a nutshell, the robust Q3 earnings of major pharma companies signals strong industry fundamentals, the volatility in pharma indices notwithstanding. Access the Pipeline Prospector Dashboard for October 2024 Newsmakers (Free Excel)

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