Market Place
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: 2962
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
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The pandemic years haven’t been great for dealmaking. Our analysis of top 10 biopharma M&A transactions shows that after a blockbuster 2019, when the combined value of top 10 biopharma M&A transactions had hit US$ 207 billion, M&A activity decreased in the next two pandemic years, with the combined value of top 10 deals dropping to US$ 97 billion in 2020 and to US$ 53 billion in 2021.In 2022, the combined value of the top 10 M&A transactions reached US$ 69 billion. However, our analysis shows that total M&As increased only marginally — from US$ 93.6 billion in 2021 to US$ 98.7 billion in 2022. In fact, the total M&As by upfront cash dropped from US$ 77.6 billion to US$ 76.3 billion. Our data does not include deals in medical devices, diagnostics and animal health.Industry watchers had predicted 2022 to be the year of M&As, as Big Pharma were expected to spend the booty amassed during the pandemic on acquisitions. But record inflation, a looming recession, the ongoing Russia-Ukraine war, volatile stock markets and some regulatory moves (such as the US Inflation Reduction Act) deterred companies from loosening their purse strings.The result was another
lackluster year for M&As. The top 10 M&A deals in 2022, selected based on upfront cash paid,
include Amgen’s Horizon takeover (US$ 27.8 billion), Pfizer’s purchase of Biohaven (US$ 11.6 billion) and Global Blood Therapeutics (US$ 5.4 billion), and BMS’ purchase of Turning Point Therapeutics (US$ 4.1 billion).View Top 100 Pharma & Biotech Deals in 2022 by Deal Size (Free Excel Available)Amgen buys Horizon in 2022’s biggest deal; picks up ChemoCentryx for US$ 3.7 bnThe year began on a sluggish note with M&A activity picking up in the second half. The biggest deal of the year — Amgen’s acquisition of Horizon Therapeutics — came in December.Last November, Horizon Therapeutics had said it
had drawn
buyout interest from players like Amgen, Sanofi and Johnson
& Johnson. Just weeks before Christmas, Amgen announced its US$ 27.8 billion takeover of Horizon. The Irish firm develops medicines for rare autoimmune and severe inflammatory diseases mostly sold in the US. Once complete, the deal will give Amgen access to Horizon’s two best-selling drugs – thyroid eye disease drug Tepezza and gout
treatment Krystexxa. Owing to the Amgen-Horizon deal, the rare
diseases therapeutic area attracted the highest amount of M&As (by deal
size). The second largest was neurology, with Pfizer taking over Biohaven and UCB
Pharma acquiring Zogenix.In August, Amgen had picked up Californian
biotech ChemoCentryx for US$ 3.7
billion to help broaden its portfolio of inflammation and kidney drugs. The deal brought ChemoCentryx’s potential blockbuster treatment for inflammatory disorders — Tavneos (avacopan) — into Amgen’s fold, along with three early-stage drug candidates that target ulcerative colitis, skin conditions and cancer.View Top 100 Pharma & Biotech Deals in 2022 by Deal Size (Free Excel Available) Pfizer acquires Biohaven for US$ 11.6 bn, Global Blood Therapeutics for
US$ 5.4 bnThe pandemic brought huge
revenue gains for Pfizer and the drug
behemoth decided to use the cash by cherry-picking companies that would
strengthen its portfolio. In May, Pfizer notched up New Haven-based Biohaven
Pharmaceuticals for US$ 11.6 billion. The deal gave
Pfizer the rights to Nurtec ODT, a leading oral migraine drug in the US and a potential blockbuster. Biohaven has six other migraine drugs in development and Pfizer expects them to top US$ 6 billion in annual sales. Biohaven is Pfizer’s largest purchase since 2016, when it bought Medivation for US$ 14 billion.Later in the year, Pfizer
acquired blood disorder drugmaker Global Blood Therapeutics (GBT) for US$ 5.4 billion with the intention of expanding its sickle cell disease portfolio. Through this buyout, Pfizer has added GBT’s approved drug, Oxbryta, along with two other sickle cell medicines – GBT601 and inclacumab (both in mid- to late-stage testing) – into its portfolio. Pfizer expects all these blood disorder drugs to contribute US$ 3 billion in peak sales.View Top 100 Pharma & Biotech Deals in 2022 by Deal Size (Free Excel Available) BMS buys Turning Point for its oncology pipeline; Takeda acquires Nimbus
LakshmiIn June, Bristol Myers Squibb acquired California-based drug developer Turning Point Therapeutics for an upfront
cash amount of US$ 4.1 billion to bolster its cancer drug pipeline. The deal gives BMS access to Turning Point’s star candidate, repotrectinib, in trials to treat non-small cell lung cancer (NSCLC) along with other advanced solid tumors. BMS expects repotrectinib to be approved in the US in the second half of 2023 and become a standard-of-care therapy for certain patients with NSCLC.In December, Takeda Pharmaceutical acquired US-based Nimbus
Lakshmi, a subsidiary of Nimbus Therapeutics that works
on TYK2 programs, for US$ 4 billion in upfront payment. Through the deal, Takeda won Nimbus’ experimental psoriasis drug TAK-279. Takeda will make two milestone payments of US$ 1 billion each to Nimbus if TAK-279 achieves annual net sales of US$ 4 billion and US$ 5 billion.View Top 100 Pharma & Biotech Deals in 2022 by Deal Size (Free Excel Available) GSK enters pneumococcal vaccine race with Affinivax deal; UCB buys ZogenixGSK – one of the world’s largest vaccine makers by sales – is set to compete for a share of the lucrative pneumococcal vaccine market with its purchase of Affinivax for a potential US$ 3.3 billion. Through the deal, the British drugmaker has gained access to the Boston-based biopharma’s 24-valent pneumococcal vaccine candidate, along with its vaccine development technology platform MAPS (Multiple Antigen Presenting System) and other assets. GSK believes the vaccine candidate will help it challenge market leader Pfizer’s blockbuster Prevnar range of vaccines.GSK also paid US$ 1.9 billion for American drug
developer Sierra Oncology and gained access to the latter’s targeted therapies for the treatment of rare forms of cancer. Sierra’s lead candidate momelotinib is being developed to address the unmet medical needs of anemic myelofibrosis patients. GSK believes the drug will work better at treating anemia than the currently approved JAK inhibitors on the market. FDA will decide on the drug’s approval by June 2023.Belgian biopharma UCB paid US$ 1.9 billion for Zogenix, expanding its
portfolio of treatments for specific and rare forms of epilepsy drugs. The
acquisition will help UCB deepen its foothold in the epilepsy market, adding to
its existing line of four products, including Vimpat and Briviact.View Top 100 Pharma & Biotech Deals in 2022 by Deal Size (Free Excel Available) Merck inks deal with Kelun Biotech; Regeneron buys rights to Sanofi’s cancer drugSeveral drugmakers announced
collaborations where milestone payments took precedence over upfront payments.
In one such deal, Merck entered into a
potential US$ 9.3 billion licensing and collaboration agreement with China’s Kelun Biotech that will give it
rights to seven early-stage antibody-drug conjugate candidates against oncology targets. Merck paid a relatively small sum
of US$ 175 million upfront to Kelun as
part of the deal.After partnering with Sanofi for seven years, Regeneron Pharmaceuticals
acquired the global rights to cancer drug Libtayo from the French
drugmaker for an upfront payment of US$ 900 million. Regeneron paid another US$ 100 million to Sanofi after FDA
approved the drug as a first-line treatment for adult patients with advanced
NSCLC (to be administered in combination with platinum-based chemotherapy). As
part of the deal, Sanofi will receive a royalty of 11 percent on worldwide net
sales of Libtayo and will get another potential US$ 100 million in
sales-related milestone payments over the next two years.View Top 100 Pharma & Biotech Deals in 2022 by Deal Size (Free Excel Available) Biocon acquires Viatris’ biosim business; Biogen sells stake in Samsung Bioepis Biocon Biologics, a subsidiary of Indian drugmaker Biocon, acquired US-based Viatris’ biosimilars business for US$ 3.34 billion. The deal expanded Biocon’s biosimilar portfolio of 20 treatments – including 11 that were with Viatris – by adding therapies for treating diabetes, tumors and autoimmune diseases. According to Biocon, Viatris’ biosimilar portfolio is expected to generate over US$ 1 billion in 2023.Samsung Biologics shelled out US$ 2.3 billion to take over partner
Biogen’s stake in Samsung Bioepis, which the duo had
set up in 2012 to develop and manufacture biosimilars. Biogen, however,
retained the commercial rights to both Byooviz (a biosimilar of Novartis’ eye drug Lucentis) and the investigational candidate SB15 (a biosimilar of Regeneron’s Eylea) along with the
biosimilars of Enbrel, Humira and Remicade.View Top 100 Pharma & Biotech Deals in 2022 by Deal Size (Free Excel Available) Our viewGlobally, M&As slowed down substantially across all industries in 2022 as companies confronted challenges such as rising interest rates, a pullback in leveraged finance, bond-market jitters and the possibility of a recession. According to Refinitiv, the total value of deals announced globally fell 37 percent last year — the biggest year-over-year percentage drop since 2001.Going by these statistics, the pharmaceutical industry didn’t fare badly. In its latest ‘Pharmaceutical & life sciences: US Deals 2023 outlook’ report, PricewaterhouseCoopers has predicted a more active 2023, with “M&As to more closely resemble prior years” with a total deal value in the US$ 225 billion to US$ 275 billion range across all sub-sectors.The new year has begun on a
positive note, with three M&A deals worth over US$ 4 billion being announced at last month’s JP Morgan Healthcare Conference. A series of patent expirations are going to put global
prescription sales at risk through 2026. Therefore, it seems plausible that the recent momentum in M&A activities will continue throughout 2023.
Impressions: 4002
https://www.pharmacompass.com/radio-compass-blog/top-100-pharma-biotech-deals-in-2022
#PharmaFlow by PHARMACOMPASS
23 Feb 2023
