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DATA COMPILATION #PharmaFlow

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New Drugs Approvals by FDA and EMA: 2020 Recap
The year 2020 was an eventful year for the pharmaceutical industry, with several companies across the world working at a feverish pace to find a treatment or a vaccine for the raging Covid-19, which has so far taken over 1.79 million lives worldwide. With countries imposing lockdowns and regulators putting on-site inspections on hold, we were expecting far lower new drug approvals in mid-2020. But our mid-2020 recap published in July, which looked at new drug approvals by the US Food and Drug Administration (FDA) and European Medical Agency (EMA), found that the FDA had approved 33 new drugs by the end of June. This put the approvals within the ballpark of the past two years.  This week, we bring you a roundup of 2020, a tumultuous year when 58 drugs (53 approvals by the Center for Drug Evaluation and Research and 5 by the Center for Biologics Evaluation and Research) bagged FDA’s new drug approvals. While this number is lower than the number of drugs approved in 2018 (62), it is higher than the number for 2019 (54). Out of this, while 23 approvals were in the field of oncology, 9 were for infectious diseases and infections, 8 for genetic diseases, 7 for neurology, 3 for immunology and 2 for gastroenterology. View New Drug Approvals in 2020 with Estimated Sales (Free Excel Available) A year marked by EUA With the pandemic raging across the world, emergency use authorizations (EUAs) dominated news headlines in 2020 — the FDA issued 10 EUAs, with the most prominent being those issued to Pfizer-BioNTech and Moderna for their Covid-19 vaccines. EMA was busy as well since they issued 75 positive opinions with Novartis leading the pack with 8, followed by Pfizer and Sanofi which received 4 each. The EUAs came with their own set of controversies. In March, the FDA had issued an EUA “for oral formulations of chloroquine phosphate and hydroxychloroquine sulfate for the treatment of” Covid-19. However, by June, FDA had revoked the EUA, as the agency determined that chloroquine and hydroxychloroquine were not likely to be effective in treating Covid-19 for the authorized uses in the EUA. Amongst treatments for Covid-19, in May the FDA authorized the emergency use of Gilead’s antiviral drug remdesivir. In our mid-2020 recap, Gilead’s remdesivir was on top of our list of top-selling drugs after it received an EUA from the FDA. In October, remdesivir became the first drug to be approved by the FDA for treatment of Covid-19 patients requiring hospitalization. While analysts predicted US$ 3.5 billion in revenue in early October, the future of this drug as a treatment for Covid-19 in hospitalized patients remains uncertain, especially in wake of results from the World Health Organization (WHO)-led Solidarity Trial that said Gilead’s remdesivir had little or no effect on the 28-day mortality or length of hospital stays for Covid-19 patients. The FDA approved remdesivir for hospitalized patients a week after the WHO results.  View New Drug Approvals in 2020 with Estimated Sales (Free Excel Available) Gilead US$ 21 billion Immunomedics acquisition Immunomedics' antibody-drug conjugate (ADC) — Trodelvy (sacituzumab govitecan-hziy) — was approved by the FDA in April this year for the treatment of adult patients with metastatic triple-negative breast cancer who have received at least two prior therapies for the disease. Such tumor types account for 15 to 20 percent of breast cancers. Trodelvy follows remdesivir in our list of FDA approved drugs in 2020 with the highest sales potential. The current forecast for Trodelvy sales is US$ 2.151 billion by 2026.  In September, Gilead made a big move and acquired biotech company Immunomedics Inc for US$ 21 billion. The transaction, which was completed in October, will strengthen Gilead’s cancer portfolio and add another potential blockbuster to it. Immunomedics plans to submit a supplemental Biologics License Application (BLA) to support full approval of Trodelvy in the US over the next quarter. According to a statement, Immunomedics is also on track to file for regulatory approval of the drug in Europe in the first half of 2021. Moreover, ongoing studies are also evaluating the potential of Trodelvy as a treatment for non-small cell lung cancer and other types of solid tumors. View New Drug Approvals in 2020 with Estimated Sales (Free Excel Available) Roche-PTC Therapeutics’ risdiplam bags approval Following Troveldy in sales potential for drugs approved by the FDA is Roche and PTC Therapeutics’ drug Evrysdi (risdiplam), the first oral medicine approved for the rare genetic disease, spinal muscular atrophy, which until four years ago had no available treatments. The approval of Evrysdi presents patients and their families with a unique choice between a one-time gene therapy, an RNA-based drug infused three times a year at the doctor’s office and a daily medicine taken at home. Roche priced the drug by patient weight, with a maximum cost of US$ 340,000 per year — substantially lesser than the competing (and approved) therapies from Biogen and Novartis. View New Drug Approvals in 2020 with Estimated Sales (Free Excel Available) Vertex’s Kaftrio bags EMA approval Earlier this year, PharmaCompass had published its compilation of sales forecasts for the new drugs approved by the FDA in 2019. The list was led by Vertex’s cystic fibrosis treatment — Trikafta — which was expected to have sales of US$ 3.935 billion by 2024.  Trikafta is a combination of ivacaftor, tezacaftor and elexacaftor and its stellar clinical data made the FDA approve the drug within three months of Vertex’s application filing and five months before FDA’s action date.  In June 2020, EMA’s CHMP adopted a positive opinion, recommending the granting of a marketing authorization for Vertex’s combination, which will be marketed as Kaftrio.  View New Drug Approvals in 2020 with Estimated Sales (Free Excel Available) A year of multiple setbacks Not everything went smoothly in 2020. In fact, the year saw several setbacks — almost 44 drugs were not granted approval by the FDA. Bristol Myers Squibb was one such company that received setbacks. As part of Bristol’s US$ 74 billion acquisition of Celgene, the New York-headquartered drug company offered Celgene shareholders Contingent Value Rights or CVRs. But to realize the US$ 9-a piece payment, approvals for three ex-Celgene drugs must meet their pre-specified deadlines. While in March, the FDA approved Bristol’s ozanimod, a sphingosine-1-phosphate receptor agonist for the treatment of relapsing multiple sclerosis, well ahead of the December 31, 2020 deadline,  in May, Bristol Myers Squibb and bluebird bio, Inc announced that they have received a Refusal to File letter from FDA regarding the Biologics License Application (BLA) for their CAR-T therapy — idecabtagene vicleucel (ide-cel) — for patients with heavily pre-treated relapsed and refractory multiple myeloma, which was submitted in March 2020. Upon preliminary review, the FDA determined that the Chemistry, Manufacturing and Control (CMC) module of the BLA requires further details to complete the review. This was followed by Bristol Myers announcing that the FDA has extended the action date by three months for the BLA for lisocabtagene maraleucel (liso-cel), a CD19-directed CAR-T therapy for the treatment of adults with relapsed or refractory (R/R) large B-cell lymphoma after at least two prior therapies. The FDA had then set the new Prescription Drug User Fee Act (PDUFA) action date as November 16, 2020. However, on that day, FDA informed the company that its review of the BLA for liso-cel will not be completed by November 16. The FDA approval of liso-cel by December 31, 2020 is one of the required remaining milestones of the CVRs issued upon the close of the Celgene acquisition in the fourth quarter of 2019. The other is FDA approval of ide-cel by March 31, 2021. FDA declines approval to Novartis’ inclisiran: Recently, there was news that the FDA declined to approve Swiss drugmaker Novartis AG’s lipid-lowering therapy, inclisiran (branded as Leqvio). The drug came to Novartis' fold through the US$ 9.7 billion acquisition of The Medicines Company last year. The drug has been cleared by the European Commission. In a statement, Novartis said the FDA has not raised any concerns related to the efficacy or safety of inclisiran. The complete response letter (CRL) is due to unresolved facility inspection-related conditions. No onsite inspection was conducted by the FDA, the company said. View New Drug Approvals in 2020 with Estimated Sales (Free Excel Available) Our view Like the drug companies, regulators across the world also worked at a frenetic pace in order to accelerate EUAs and drug approvals, especially for treatments and vaccines for Covid-19. Apart from Covid-19, the year saw wider adoption and approvals for cell and gene therapies along with approvals of several innovative medicines like relugolix (the first oral gonadotropin-releasing hormone receptor antagonist for the treatment of adult patients with advanced prostate cancer), berotralstat the first oral once daily plasma kallikrein inhibitor to prevent attacks of hereditary angioedema in adults and pediatric patients 12 years and older, lumasiran (an HAO1-directed small interfering ribonucleic acid indicated for the treatment of primary hyperoxaluria type 1 to lower urinary oxalate levels in pediatric and adult patients) and osilodrostat for the treatment of adults with Cushing’s disease). Moreover, there were several interesting ‘non-Covid’ medical breakthroughs that took place during 2020. One such breakthrough is a single pill that combines four medications meant to lower blood pressure and cholesterol and aspirin that was found to cut the risk of heart disease. While the agility shown by pharma companies and regulators was undoubtedly quite impressive, with many countries granting EUA to vaccines for Covid-19, the ongoing pandemic did put mankind in a bind for several months. And if the words of the WHO director general Tedros Adhanom Ghebreyesus are to be believed, the coronavirus crisis will not be the last pandemic and attempts to improve human health are “doomed” without tackling climate change and animal welfare. If that really proves to be the case, the pharma industry has a lot to learn from this pandemic, and science has a lot to demonstrate if mankind is to emerge unscathed from such adversities. View New Drug Approvals in 2020 with Estimated Sales (Free Excel Available)  

Impressions: 7035

https://www.pharmacompass.com/radio-compass-blog/new-drugs-approvals-by-fda-and-ema-2020-recap

#PharmaFlow by PHARMACOMPASS
31 Dec 2020

STOCK RECAP #PipelineProspector

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Pipeline Prospector Oct 2023: Disappointing Q3 results, attacks on Israel, Gaza drag pharma indices down further
October was a gloomy month that saw Palestinian militant group Hamas attack Israel in the first week, resulting in the killing of hundreds of innocent people. Israel retaliated, formally declaring a war on Hamas. Another raging war, after the ongoing Russia-Ukraine conflict, doesn’t augur well for the planet, let alone humanity, economies and industries.The pharma indices reacted to the bloodshed, as also to the disappointing third quarter (Q3) results posted by some drugmakers. The Nasdaq Biotechnology Index (NBI) was down 8 percent, at US$ 3,637.05 while the SPDR S&P Biotech ETF (XBI) was down 12 percent, at US$ 64.12, and the S&P Biotechnology Select Industry Index (SPSIBI) was down by 9 percent, at US$ 4,991.31.On the upside, the month saw several large deals. The biggest deal of the month was Roche’s acquisition of Telavant from Roivant for US$ 7.1 billion. BMS announced the acquisition of Mirati Therapeutics for US$ 4.8 billion. Through this acquisition, BMS will gain access to the US Food and Drug Administration (FDA) approved lung cancer drug Krazati (adagrasib). And Eli Lilly expanded its oncology capabilities by acquiring Point Biopharma for US$ 1.4 billion.Access the Pipeline Prospector Dashboard for October 2023 Newsmakers (Free Excel)Sanofi, Takeda cut 2023 profit forecasts; AbbVie, BMS post drop in Q3 salesSeveral drugmakers announced their Q3 2023 results last month. Sanofi (stock down by 15 percent in October) reported a 4.1 percent drop in Q3 sales, at €11.9 billion (US$ 12.5 billion). What was worse, the French drugmaker lost €20 billion (US$ 21 billion) in market capitalization soon after announcing these results as it had abandoned its 2025 profit target of 32 percent. Instead of chasing the lofty target, Sanofi has decided to focus on “long-term profitability”, and increase spending on immunology and inflammation drug development. Sales of Sanofi’s multiple sclerosis drug Aubagio dropped by a whopping 60.5 percent to €199 million (US$ 210.2 million) primarily due to competition from generics in the US market. Takeda (down by 7 percent) slashed its full-year profit forecast by 36 percent to 225 billion yen (US$ 1.5 billion) as it contends with disappointments in its development pipeline and the loss of patent protection in key products.BMS (down by 10 percent) reported Q3 revenue of US$ 11 billion, a 2 percent decrease from the same quarter last year, mainly due to 41 percent decline in sales of its cancer drug Revlimid.AbbVie (down by 5 percent) reported Q3 net revenues of US$ 13.92 billion, marking a 6 percent decrease over Q3 2022. This drop is attributed in part to the launch of Humira biosimilars, which have caused a 36.2 percent reduction in the immunology drug’s net revenues. Additionally, AbbVie’s blood cancer drug Imbruvica’s net revenues have dropped by 20 percent.Access the Pipeline Prospector Dashboard for October 2023 Newsmakers (Free Excel) Low Covid sales hit Pfizer, BioNTech, Roche stocks; Pfizer’s Q3 sales dip by 42%Pfizer (down by 9 percent) saw a 42 percent decline in its Q3 revenues — at US$ 13.23 billion — primarily due to lower sales of its Covid products. While sales of its mRNA vaccine Comirnaty fell by 70 percent (at US$ 1.3 billion), its Covid drug Paxlovid saw a 97 percent drop in sales over Q3 2022.Pfizer has significantly reduced its full-year revenue forecast and also plans to save US$ 3.5 billion through job cuts and cost reductions. Its partner for Comirnaty, BioNTech, is yet to announce its Q3 results, but its stock fell by 13 percent during October.Roche’s Q3 sales also slipped by 3 percent to CHF 14.3 billion (US$ 15.9 billion) owing to a slump in Covid sales. The Swiss drugmaker has predicted a decline in 2023 sales of Covid products at about CHF 4.5 billion (US$ 4.99 billion). Its previous estimate was CHF 5 billion (US$ 5.5 billion). Roche’s stock was down by 6 percent last month.Access the Pipeline Prospector Dashboard for October 2023 Newsmakers (Free Excel) Merck, Novo raise 2023 guidance; Ozempic, Wegovy boost Novo salesAmong the gainers were mega-cap drugmakers Merck (stock up by 1 percent in October) and Novo Nordisk (up by 4 percent). Merck has reported higher-than-expected third-quarter results on strong demand for its Covid-19 treatment, Lagevrio (molnupiravir), cancer med Keytruda and HPV vaccine Gardasil. As a result, Merck has revised its 2023 sales upwards, in the range of US$ 59.7 billion to US$ 60.2 billion (from US$ 58.6 billion to US$ 59.6 billion).Novo Nordisk’s popular diabetes med Ozempic and weight loss med Wegovy are boosting the company’s sales. Ergo, the company expects full-year sales to grow between 32 and 38 percent at constant exchange rates. Moreover, Novo has acquired ocedurenone, a drug in late-stage trial, from KBP Biosciences for up to US$ 1.3 billion. Ocedurenone is an oral treatment for uncontrolled hypertension with potential applications in cardiovascular and kidney disease.Access the Pipeline Prospector Dashboard for October 2023 Newsmakers (Free Excel) Astra, Ventyx stocks hit by disappointing trial data; Lilly, Vertex post gainsAstraZeneca’s stock was hit by disappointing results from a late-stage trial of its experimental lung cancer drug — datopotamab deruxtecan. Astra’s stock was down by 6 percent in October and its partner in developing the drug, Daiichi Sankyo’s stock fell by 4 percent.Similarly, Ventyx Biosciences (down by 56 percent) announced positive results from a phase 2 trial  in patients with moderate-to-severely active ulcerative colitis, but investors were unimpressed due to doubts about whether the candidate has an edge over rival Pfizer’s etrasimod and BMS’ Zeposia. Akero Therapeutics’ (down by 76 percent) nonalcoholic steatohepatitis (NASH) candidate, efruxifermin, failed mid-stage trial as it didn’t show any significant benefit for patients with cirrhosis caused by NASH, the most advanced and life-threatening stage of the fatty liver disease. Similarly, Sarepta Therapeutics’ treatment for Duchenne muscular dystrophy (a muscle-wasting disease) — Elevidys — failed a phase 3 study, taking its stock down by 44 percent in October.In a positive clinical trial news, Lilly’s Crohn’s disease experimental drug mirikizumab met the main goals of a phase 3 study. Similarly, Vertex (up by 4 percent) posted encouraging positive clinical trial results of VX-880 to treat type 1 diabetes.Meanwhile, FDA issued a complete response letter (CRL) to Alnylam Pharmaceuticals’ siRNA therapy Onpattro (patisiran) for the treatment of cardiomyopathy of transthyretin-mediated amyloidosis. It stated that Alnylam (down by 15 percent) has not provided enough evidence of the therapy’s benefit in the proposed indication.Access the Pipeline Prospector Dashboard for October 2023 Newsmakers (Free Excel) Our viewThe biopharma industry is eagerly awaiting the approval of several experimental drugs in Q4 2023, including Italfarmaco’s givinostat for Duchenne muscular dystrophy (DMD), and Valneva’s single-shot chikungunya vaccine VLA1553.We are already seeing the market inch upwards. Let’s hope the exciting pipeline and new drug approvals are able to pull pharma indices out of the doldrums.Access the Pipeline Prospector Dashboard for October 2023 Newsmakers (Free Excel) 

Impressions: 2285

https://www.pharmacompass.com/pipeline-prospector-blog/disappointing-q3-results-attacks-on-israel-gaza-drag-pharma-indices-down-further

#PharmaFlow by PHARMACOMPASS
02 Nov 2023

NEWS #PharmaBuzz

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https://www.expresspharma.in/s1p-receptor-modulators-face-uncertain-future-in-crohns-disease-amid-trial-setbacks/

EXPRESSPHARMA
14 Oct 2024

https://www.businesswire.com/news/home/20240917429806/en

BUSINESSWIRE
18 Sep 2024

https://www.pharmacompass.com/pdf/news/fda-confirms-para-iv-patent-litigation-for-ozanimod-hcl-zeposia-capsules-92708.pdf

FDA
05 Jun 2024

https://www.reuters.com/business/healthcare-pharmaceuticals/bristol-myers-bowel-disease-drug-fails-meet-main-goal-late-stage-study-2024-03-28/

REUTERS
29 Mar 2024

https://www.businesswire.com/news/home/20240327817166/en

BUSINESSWIRE
28 Mar 2024

https://www.businesswire.com/news/home/20231010049123/en

BUSINESSWIRE
11 Oct 2023