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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: 3071

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 Jan 2020: Deals and developments in pharma and biotech
We at PharmaCompass are pleased to announce the launch of Pipeline Prospector, in collaboration with SCORR Marketing, a leading health science marketing agency. The Pipeline Prospector is a free-access database of global drug development deals and updates designed for executives in the drug development industry. Our database will be updated every month. The first of this two-part rollout of the initial version of the Pipeline Prospector delivers access to a database of current deals and developments. In the second phase, we will add pipeline data to provide more in-depth therapeutic information. Access the Pipeline Prospector Dashboard for All Deals & Development Updates We begin 2020 with a compilation of all the major deals and developments which were announced in January. Dealmakers at JPMorgan Historically, January is the month of the annual JPMorgan Healthcare Conference in San Francisco — a platform where drug majors tend to announce significant deals and strategic shifts. Last year witnessed the US$ 74 billion acquisition of New Jersey-based cancer drug company Celgene by Bristol-Myers Squibb. After factoring in debt, the value of the deal ballooned to about US$ 95 billion, which according to data compiled by Refinitiv, was the largest healthcare deal on record. This year started on a muted note with Eli Lilly’s all-cash acquisition of California-based Dermira Inc being one of the only major deals. The acquisition will allow Eli Lilly to expand its immunology pipeline with a late-stage treatment for atopic dermatitis. The acquisition gives Lilly two key Dermira assets — lebrikizumab and Qbrexza (glycopyrronium) cloth. Lebrikizumab is a novel, investigational, monoclonal antibody designed to bind IL-13, which is believed to be a driver of signs and symptoms of atopic dermatitis. It is currently in two Phase III trials for the treatment of moderate-to-severe atopic dermatitis in adolescent and adult patients, aged 12 years and older. In late 2019, FDA granted fast-track designation to lebrikizumab, and analysts are of the view that the drug could be a challenger to Dupixent, a skin drug made by Regeneron and Sanofi that is expected to bring in over US$ 11 billion in annual sales.  Access the Pipeline Prospector Dashboard for All Deals & Development Updates At the JPMorgan conference, Incyte and MorphoSys announced a collaboration and licensing agreement to further develop and commercialize MorphoSys’ proprietary anti-CD19 antibody tafasitamab (MOR208) globally. Tafasitamab is an Fc-engineered antibody against CD19 currently in clinical development for the treatment of B cell malignancies. MorphoSys and Incyte will co-commercialize tafasitamab in the US, while Incyte has exclusive commercialization rights outside of the US. As per the agreement, MorphoSys will receive an upfront payment of US$ 750 million. In addition, Incyte will make an equity investment into MorphoSys to the tune of US$ 150 million. Depending on the achievement of certain developmental, regulatory and commercial milestones, MorphoSys will be eligible to receive milestone payments amounting to nearly US$ 1.1 billion. MorphoSys will also receive tiered royalties on ex-US net sales of tafasitamab in a mid-teens to mid-twenties percentage range of net sales. An approval for tafasitimab could come as soon as mid-2020, according to MorphoSys, which submitted the drug to the US Food and Drug Administration in late 2019. Around the same time as these deals were announced, Biogen placed another bet on Alzheimer’s R&D by paying Pfizer US$ 75 million upfront for a candidate currently in Phase I trials. Access the Pipeline Prospector Dashboard for All Deals & Development Updates Amgen’s China bet January also saw the completion of two previously announced US$ 2.7 billion deals. As China, the world’s second largest pharmaceutical market, continues to remain a key growth area for Big Pharma, Amgen completed its acquisition of a 20.5 percent stake in the Chinese biotechnology firm Beigene worth approximately US$ 2.7 billion. The deal will give Amgen’s cancer drugs greater exposure to the Chinese market and also allow the firm to profit if BeiGene’s pipeline of molecularly targeted and immuno-oncology products prove to be effective and ready for commercialization. A month after Amgen had announced its investment in BeiGene, the FDA approved BeiGene’s cancer therapy zanubrutinib (branded as Brukinsa) months ahead of schedule. This was the first-ever FDA approval for a cancer therapy developed by a Chinese biotech. Access the Pipeline Prospector Dashboard for All Deals & Development Updates Merck’s oncology focus US-headquartered Merck, which is known as MSD (short for Merck Sharp & Dohme) outside the United States and Canada, completed its acquisition of ArQule through its subsidiary for US$ 2.7 billion. ArQule focusses on kinase inhibitor discovery and development for the treatment of patients with cancer and other diseases. The deal gives Merck access to ArQule’s experimental treatment ARQ 531 — a novel, oral Bruton’s tyrosine kinase (BTK) inhibitor currently in a Phase 2 dose expansion study for the treatment of B-cell malignancies. In January, Merck also announced an alliance with Japanese drugmakers Taiho Pharmaceutical and Astex Pharmaceuticals (a wholly owned subsidiary of Otsuka Pharmaceutical). The American drugmaker said it is willing to invest up to US$ 2.5 billion in order to gain access to small-molecule inhibitors against several drug targets, including the KRAS oncogene, from Taiho and Astex. Access the Pipeline Prospector Dashboard for All Deals & Development Updates According to a statement, Taiho, Astex and Merck will combine preclinical candidates and their data “with knowledge and expertise from their respective research programs.” In exchange for giving Merck an exclusive global license to their small-molecule inhibitor candidates, Taiho and Astex will get US$ 50 million upfront, with the US$ 2.5 billion “contingent upon the achievement of preclinical, clinical, regulatory and sales milestones for multiple products arising from the agreement, as well as tiered royalties on sales.” “Merck will fund research and development and will be responsible for commercialization of products globally,” the statement said. Taiho has retained co-commercialization rights in Japan and an option to promote the drugs (coming out of this alliance) in specific areas of South East Asia. KRAS is short for the gene Kirsten rat sarcoma viral oncogene homolog, which makes a protein (KRAS protein) that is critical in promoting cell growth and survival in non-cancerous cells. Cancers driven by KRAS mutations are both common and deadly. The year 2019 was a big one for KRAS, when several players — such as Amgen, Boehringer Ingelheim, Eli Lilly and Mirati — had reported progress in the field. For instance, Amgen had reported encouraging anti-tumor activity with its AMG 510 candidate, which targets a KRAS mutation known as G12C. In September last year, Boehringer Ingelheim (BI) had licensed an MEK inhibitor from Indian drugmaker Lupin that it intends to pair with its lineup of KRAS inhibitors. BI had also entered into a partnership with MD Anderson Cancer Centre in the US for another KRAS inhibitor. Access the Pipeline Prospector Dashboard for All Deals & Development Updates Acceleron’s shares skyrocket The share price of Boston-based Acceleron almost doubled in the month of January when new Phase 2 data on the success of an experimental heart drug, sotatercept, found a statistically significant decrease in pulmonary vascular resistance in patients with pulmonary arterial hypertension(PAH). Pulmonary arterial hypertension (PAH) is a rare, progressive disorder characterized by high blood pressure (hypertension) in the arteries of the lungs (pulmonary artery) for no apparent reason. The pulmonary arteries are the blood vessels that carry blood from the right side of the heart through the lungs. In November 2019, the FDA approved Acceleron’s luspatercept (brand name Reblozyl) for the treatment of anemia among patients with beta-thalassemia who require regular red blood cell transfusions. The approval provides patients with a therapy that, for the first time, will help decrease the number of blood transfusions. Access the Pipeline Prospector Dashboard for All Deals & Development Updates Setback for France’s Ipsen and Nektar Therapeutics French drugmaker Ipsen announced it was pausing a phase III trial of palovarotene, an experimental rare bone disease drug, after an interim analysis found it is unlikely to meet its primary efficacy endpoint. The decision to pause the trial is based on the results of a futility analysis reviewed by the Independent Data Monitoring Committee (IDMC). The announcement marked yet another blow for palovarotene, which had run into safety and efficacy problems since Ipsen acquired it in a US$ 1.3 billion (€1.1 billion) takeover of Clementia Pharmaceuticals. In December, a safety concern prompted the FDA to halt dosing in children. At the time of acquiring Clementia last year, David Meek, the then CEO of Ipsen, had called the drug “a largely derisked asset”. Since then, palovarotene has been the subject of a safety signal that led the FDA to stop the trial in children, a key population. The drug has now been rocked by an analysis that questions its efficacy. Access the Pipeline Prospector Dashboard for All Deals & Development Updates Last month, we also witnessed a major setback for Nektar Therapeutics as a joint FDA advisory committee voted 27-0 against the approval of oxycodegol for chronic low back pain. Oxycodegol’s key selling point was that its central nervous system absorption rate was much slower than that of the other often abused opioid drugs.  The focus on drug development is constantly evolving and the Pipeline Prospector, our free access database of global drug development deals and development updates, is designed to provide the insights necessary for professionals to drive their business forward. Email us at support@pipelineprospector.com to learn more. Access the Pipeline Prospector Dashboard for All Deals & Development Updates  

Impressions: 3780

https://www.pharmacompass.com/pipeline-prospector-blog/pipeline-prospector-jan-2020-deals-and-developments-in-pharma-and-biotech

#Phispers by PHARMACOMPASS
13 Feb 2020

NEWS #PharmaBuzz

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https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-approves-ipsens-rare-bone-disorder-drug-2023-08-16/

REUTERS
17 Aug 2023

https://www.reuters.com/business/healthcare-pharmaceuticals/ipsen-says-european-commission-rejects-marketing-authorization-bone-disorder-2023-07-19/

REUTERS
19 Jul 2023

https://endpts.com/fda-adcomm-votes-in-favor-of-ipsens-fibrodysplasia-drug/

Katherine Lewin ENDPTS
29 Jun 2023

https://www.globenewswire.com/news-release/2023/06/29/2696672/0/en/Ipsen-announces-positive-outcome-of-FDA-Advisory-Committee-on-investigational-palovarotene-for-fibrodysplasia-ossificans-progressiva.html

GLOBENEWSWIRE
28 Jun 2023

https://endpts.com/fda-reviewers-on-ipsens-potential-fibrodysplasia-drug-post-hoc-analysis-may-be-enough/

Zachary Brennan ENDPTS
27 Jun 2023

https://www.globenewswire.com/news-release/2023/05/26/2677082/0/en/Ipsen-receives-CHMP-negative-opinion-following-re-examination-of-potential-first-FOP-treatment-in-the-E-U.html

GLOBENEWSWIRE
26 May 2023