Generic drugs play a crucial role in providing access to life-saving drugs at affordable prices. To that end, drugmakers submit Drug Master Files (DMFs) or their ‘recipes for making generics’ to the US Food and Drug Administration (FDA) for review. Of these, Type II DMFs involve active pharmaceutical ingredients (APIs) for both branded and generic drugs.PharmaCompass has been reviewing Type II
submissions for several years now. We have noticed that these filings have been
increasing in recent years. There has been a 33.5 percent
increase in Type II DMF submissions since Q1 2018 (when 176 Type II DMFs had
been submitted). However, at 235, the first quarter (Q1) of 2024 saw only a 1.3
percent increase in DMF submissions over Q1 2023. In Q1 2023, DMF submissions
had increased by 21.5 percent (over Q1 2022).Of the 235 Type II submissions received by the FDA in Q1 of this
year, only 35 (or around 15 percent) had their review completed under the Generic Drug User Fee Act (GDUFA). The total number of reviews completed by the US federal agency so far this year is 129.In all, FDA received 353 Type II, III, IV, and V DMF submissions,
compared to 291 in Q1 2023, an increase of 21.3 percent.View FDA DMF Filings in Q1 2024 (Power BI Dashboard, Free Excel Available)DMF submissions from India
dip 1.8%; China witnesses 42% rise in filings As has been the trend, India (dubbed as the pharmacy of the world) and China filed the maximum DMF submissions, with 107 and 101 submissions respectively. However, DMFs filed by Indian companies saw a marginal dip of 1.8 percent in Q1 2024 — in Q1 2023, Indian companies had filed 109 Type II DMFs. Meanwhile, DMF filings by Chinese drugmakers increased 42.2 percent in Q1 2024, up from 71 in Q1 2023.India’s MSN Laboratories was the clear leader with 15 DMF submissions to the FDA, while China’s Sichuan Elixir
Pharmaceutical came a distant second with nine submissions. Indian drugmakers Aurobindo Pharma and Global Calcium (with seven submissions each) were tied at third position. There were two players at the fifth spot — India’s Dr. Reddy’s Laboratories and China’s Zhejiang Jingsheng
Pharmaceutical — as both these drugmakers registered six submissions each.View FDA DMF Filings in Q1 2024 (Power BI Dashboard, Free Excel Available) Diabetes, cancer drugs emerge
as hot molecules in Q1 2024Approved in June 2020, triheptanoin is the first and only FDA-approved treatment for
children and adults with long-chain fatty acid oxidation disorders (LC-FAOD).
At five, triheptanoin saw the maximum DMF filings.
Four of those applications have already been positively reviewed.Anti-diabetic drug sitagliptin phosphate
monohydrate saw four DMF filings. Empagliflozin, the API found in Boehringer Ingelheim and Eli Lilly’s diabetes drug Jardiance, drew three DMF filings in Q1 2024.In the US, Jazz Pharmaceuticals’ Zepzelca (lurbinectedin) is indicated for the treatment of adult patients with metastatic small cell lung cancer (SCLC). Three DMF applications were submitted for lurbinectedin. Similarly, ruxolitinib phosphate, used for the treatment of
patients with intermediate or high-risk myelofibrosis, also received three DMF
applications.Semaglutide — the glucagon-like peptide-1 receptor agonist that catapulted Novo Nordisk to the position of the most valuable public company in Europe — received three DMF filings. Semaglutide’s arch rival, tirzepatide, also drew three DMF filings.AstraZeneca’s Brilinta (ticagrelor) plus aspirin is currently approved in
over 115 countries for the prevention of stroke, heart attack, and other events
in adults with acute coronary syndrome (ACS). Ticagrelor received three DMF
submissions. Vonoprazan fumarate, a first-in-class potassium-competitive acid
blocker to treat acid-related diseases, also received three DMF submissions.View FDA DMF Filings in Q1 2024 (Power BI Dashboard, Free Excel Available) Eighteen DMFs filed for first
time in Q1 2024 During the first quarter of this year, 14 drugs saw first time filing of DMFs. Together, these 14 drugs attracted 18 DMF filings with tirzepatide topping the list with three submissions from Chinese companies.The 14 drugs that saw first time filing were clascoterone, capmatinib hydrochloride, niraparib tosylate
monohydrate, vibegron, cabozantinib sulfate,
ruxolitinib hemifumarate, ripretinib, ruxolitinib, ruxolitinib mesylate, tucatinib hemiethanolate, tecovirimat, tirzepatide, tolvaptan povidone, and azilsartan.View FDA DMF Filings in Q1 2024 (Power BI Dashboard, Free Excel Available) Our viewUntil 2020, DMF submissions by Indian companies used to be double those of Chinese and American
firms put together. In 2022, DMF filings from India began to dip, while
submissions by Chinese companies began to rise. In the first half of 2023,
there was a 46.5 percent rise in DMF submissions from China. The gap between
DMF filings by India and China has narrowed down considerably this year. The
reasons behind this phenomenon may be multiple. But it definitely implies that
America will find it increasingly difficult to reduce its reliance on China.
Impressions: 2437
The pharma industry clearly recalibrated itself in 2023, turning its focus away from Covid and onto two of the biggest threats to human health – obesity and cancer. The top lines of the major pharma companies reflect this shift in focus.We
always knew that Pfizer’s record US$ 100 billion revenue for 2022 wasn’t sustainable. Even though Pfizer’s 2023 sales were lower by nearly 42 percent against its 2022 sales, the New York-headquartered drugmaker managed to retain its pole position. The two main reasons behind its ‘top of the charts’ sales of US$ 58.5 billion were Pfizer’s record nine new molecular entity approvals by the US
Food and Drug Administration (FDA) and the launch of its vaccine for
respiratory syncytial virus (RSV).Johnson & Johnson came second with sales of US$ 54.8
billion (excluding its consumer business and MedTech units). AbbVie took bronze despite Humira being subject to biosimilar competition
and Merck maintained its fourth position. Roche nabbed the fifth position from Novartis (which stood sixth). Bristol Myers Squibb maintained its position at seven, as did
AstraZeneca (eighth) and Sanofi (ninth). And Eli Lilly bumped into the tenth spot, knocking out
GSK.View Our Interactive Dashboard on Top Drugs in 2023 by Sales (Free Excel Available)Keytruda, Eliquis, Humira top charts; Novo’s Ozempic debuts top 10 list at number fourMerck’s Keytruda became the number one selling drug in the world, a position that was held
by AbbVie’s Humira for long, and Pfizer’s Comirnaty in the Covid years. This
oncology drug raked in a whopping US$ 25 billion, with sales increasing 19 percent last
year. In fact, Keytruda accounted for 46.7 percent of Merck’s pharmaceutical sales, which grew 3 percent in 2023 to US$ 53.6 billion.At number two was
Pfizer and BMS’ anticoagulant Eliquis — it posted global sales of US$
18.95 billion (marking a
growth of 4
percent on 2022 sales). With
competition from generics, Humira’s sales fell by 32 percent to US$ 14.5 billion. As a result, this
blockbuster anti-rheumatic drug fell to the third rank.The
fourth spot was taken up by Novo Nordisk’s Ozempic, the wonder drug that treats type 2
diabetes. Gilead’s Biktarvy, a med that treats HIV-1, saw sales jump 14 percent — from US$ 10.39 billion posted in 2022 to US$ 11.85 billion last year. This way, Biktarvy emerged as
the fifth largest selling drug of 2023.At
number six was Sanofi and Regeneron’s Dupixent. This allergic diseases med posted
11-figure sales in 2023, netting € 10.72 billion (US$ 11.59 billion) globally, a growth of 34 percent over
2022 numbers.At
number seven was J&J’s biggest blockbuster immunology drug Stelara that raked in US$ 11.3 billion in 2023. Coming a close eighth was Pfizer-BioNTech’s Covid-19 vaccine Comirnaty — its sales fell by over 70 percent to US$ 11.22 billion in 2023. At the ninth spot was Lilly and Boehringer’s diabetes drug Jardiance that saw a 27.7 percent increase in
total global sales at US$ 10.6 billion. And rounding off the list at number 10
is BMS’s Opdivo, a Keytruda rival. Opdivo hauled in US$ 10 billion
in total global sales in 2023, a year-on-year increase of 8 percent.View Our Interactive Dashboard on Top Drugs in 2023 by Sales (Free Excel Available)Driven by diabetes, obesity care meds,
Novo, Lilly post double-digit sales growthDemand
for diabetes and new weight-loss drugs catapulted Novo Nordisk to emerge as the most valuable public
company in Europe. Its net sales zoomed 31 percent to DKK 232.3 billion (US$ 33.75 billion) compared to DKK 177
billion (US$ 25.8 billion) in 2022. Net profit jumped 51 percent to DKK 83.68 billion (US$ 12.51 billion) in 2023 from DKK
55.5 billion (US$ 8.32
billion) in 2022 — the highest annual profit for the Danish drugmaker in over three decades.The
growth was driven by Ozempic, whose sales spiked 60 percent in 2023
to DKK 95.7 billion (US$ 13.91 billion), from DKK 59.8 billion (US$ 8.71 billion) the
year before.Rival
Eli Lilly’s revenue grew 20 percent in 2023 to US$ 34.1 billion from US$ 28.5 billion in 2022. Mounjaro turned out to be a star for the
Indianapolis drugmaker with its sales rocketing 970 percent in 2023 to US$ 5.16 billion. FDA also approved it to treat obesity
under the brand name Zepbound in November, which brought in additional
revenues of US$ 176 million.View Our Interactive Dashboard on Top Drugs in 2023 by Sales (Free Excel Available) GSK’s RSV jab makes strong debut; AbbVie’s immunology drugs post steep growthGSK’s Arexvy was the first RSV vaccine approved by the FDA. It made a strong debut — Arexvy contributed £ 1.2 billion (US$ 1.5 billion) to GSK’s sales in just four months.AbbVie posted another solid financial year.
Though the drop in Humira revenue was offset by two newer
immunology blockbuster drugs, Skyrizi and Rinvoq, the Illinois-headquartered drugmaker
did posted a marginal decrease in revenue of 6.4 percent to US$ 54.3 billion. However, revenue from Skyrizi soared 50 percent to US$ 7.8 billion,
while Rinvoq’s sales increased 57 percent to US$ 4
billion. AbbVie expects a combined US$ 16 billion from Skyrizi (US$ 10.5 billion) and Rinvoq (US$ 5.5 billion) sales in 2024. BMS attributed its 2 percent decrease in
revenue (of US$ 45 billion) to lower sales of Revlimid in the US due to competition from
generics. Sales of the multiple myeloma treatment dropped 39 percent to US$ 6.1
billion. Ophthalmology drug Eylea saw a drop in sales of
4 percent, at US$ 9.21 billion (from US$ 9.65 billion), as competition from Roche’s Vabysmo triggered a price cut by Regeneron. Vabysmo saw sales balloon 324 percent
from CHF 591 million (US$ 685.56 million) to CHF 2.4 billion (US$ 2.78 billion) in 2023.View Our Interactive Dashboard on Top Drugs in 2023 by Sales (Free Excel Available) Our viewAccording
to data analytics company GlobalData, GLP-1 agonist drugs (such as Ozempic and Mounjaro that treat type 2 diabetes) are slated to overtake PD-1 antagonists (such as oncology drugs
Keytruda and Opdivo) as the top-selling drugs on the market
in 2024. It estimates a robust compounded annual growth rate (CAGR) of 19.2
percent from 2023 to 2029 for GLP-1 drugs that seem to have more benefits
besides bringing down blood sugar levels (such as weight management, benefits
to the heart etc).The
market size for GLP-1 is likely to increase to US$ 105 billion by 2029. In
contrast, the data firm projects a CAGR of 4.7 percent in the PD-1 antagonist
market, with its market size projected to be around US$ 51 billion in 2029.
Given these projections, we are likely to see more movers and shakers in our
top 10 drug list this year.
Impressions: 3301
Every week,
PharmaCompass compiles important developments in the world of pharmaceuticals
and brings a compilation to you in the form of Phispers. Of the hundreds of
stories we carried in 2023, here are the top 10 stories, including some trends
and updates.I. Pfizer buys Seagen for US$ 43 billion to
bolster its oncology portfolioIn March, Pfizer said it is acquiring cancer treatment specialist Seagen for US$ 43 billion. Seagen is a pioneer in antibody-drug conjugates
(ADCs), or drugs that work like “guided missiles” to destroy cancer cells while sparing healthy cells. Another important deal in the field of ADCs took place in December when AbbVie picked up ImmunoGen for US$ 10.1 billion, giving it access to Elahere (mirvetuximab soravtansine-gynx), an ADC approved for platinum-resistant ovarian
cancer. Elahere is expected to achieve blockbuster status by 2030. II.
Merck, BMS, trade bodies, sue US government over IRA negotiationsIn June, Merck filed a lawsuit against the US government seeking to block Medicare from negotiating lower
prescription drug prices under the Inflation Reduction Act (IRA). Days later,
the US Chamber of Commerce, one of the most influential trade groups in the US, filed a separate lawsuit, arguing
that the negotiations violated drugmakers’ constitutional rights and granted
excessive control over prices to the government. They were joined by Bristol Myers Squibb (BMS) and lobby group PhRMA. Drugmakers and
the Biden administration appeared to be at each other’s throats. In December, the White House identified 48 drugs whose prices spiked faster than inflation in Q4.
These drugs may be subject to rebates starting January 2024. Biden
Administration also announced it is setting a new “march-in” policy that allows the government to seize medicine patents held by drugmakers for therapies whose
development was taxpayer-funded, if it believes they are not “reasonably available and affordable.”III. US, UK approve Lilly’s Mounjaro for weight management; to be
sold as ZepboundIn November, drug
regulators in the US and the United Kingdom approved Eli Lilly's Mounjaro (tirzepatide) for weight management, to be sold under the brand name Zepbound. The drug will pose strong competition
to Novo Nordisk’s Wegovy in a market that's expected to reach US$ 100 billion by the end of
the decade.IV. Novo, Lilly plan capacity expansions for
weight loss drugsBoth Novo Nordisk and Eli Lilly announced expanding their manufacturing capacities in
order to capitalize on the burgeoning market for weight loss drugs. Novo is
investing over DKK 42 billion (US$ 6 billion) in Kalundborg (Denmark), US$ 2.3 billion to expand its site in Chartres (France) and over € 2 billion (US$ 2.18 billion) in Dublin (Ireland) to boost production of its blockbuster diabetes
and weight-loss drugs, including Ozempic and Wegovy (both semaglutide). Similarly, Eli Lilly had announced a US$ 2.5 billion manufacturing facility in Germany in November to address the demand for its new obesity and diabetes therapies.V. FDA finds violations at Global Pharma’s eye drops plant in India; issues Form
483In April, FDA
found several violations in manufacturing processes and sterilization methods
used by India-based Global Pharma for its EzriCare Artificial Tears Eye Drop, which has been linked to
68 cases of eye infection in the US, including eight cases of vision loss and three deaths.VI. ‘Intas India staff tore documents, threw acid to destroy evidence’, notes FDAIn January, FDA
issued a Form 483 with 11 observations to Intas Pharma’s
drug manufacturing facility in Ahmedabad (Gujarat, India). A team of three FDA
drug regulators conducted an inspection of the manufacturing facility from
November 22 to December 2, 2022. The 36-page report issued by the FDA has alleged that employees at the
facility had destroyed documents related to manufacturing practices by tearing
them into pieces and disposing them inside the quality control lab and scrap
areas. Acid was used to destroy evidence, notes FDA.VII. GSK overtakes Pfizer in bagging first FDA
approval for RSV vaccineIn May, FDA
approved GSK’s
respiratory syncytial virus (RSV) vaccine for people aged 60 and above. Arexvy is the first RSV vaccine to be approved in the US
for the common condition that can be fatal for the elderly. Later that month,
Pfizer’s RSV vaccine Abrysvo also got approved. In July, Sanofi-AstraZeneca’s
RSV antibody therapy, Beyfortus (nirsevimab-alip), received approval from the FDA. It is a long-acting treatment that can be given
once per season. The approval is specifically developed for newborns and
infants.VIII. UK authorizes gene therapy Casgevy for
blood disorders, US follows suit In November, Britain’s Medicines and Healthcare products Regulatory Agency was first off the block in authorizing CRISPR Therapeutics and Vertex Pharmaceuticals’ Casgevy, a therapy that seeks to cure two blood disorders — sickle-cell disease (SCD) and β-thalassemia. The therapy is based on gene editing technology that had won its scientists the Nobel Prize in Chemistry in 2020.Less than a month
later, FDA not only approved Casgevy (exagamglogene autotemcel) for SCD, but also approved bluebird bio’s
Lyfgenia (lovotibeglogene autotemcel) for the treatment of SCD in patients aged
12 and older who have a history of vaso-occlusive events (when tissues become
deprived of oxygen).IX. Leqembi becomes first med to bag full
approval to treat Alzheimer’sEisai and Biogen’s Alzheimer’s drug Leqembi (lecanemab) had won FDA’s accelerated approval in January. It treats patients who are in the earliest
stages of the neurodegenerative disease. In July, it became the first treatment to receive full FDA approval to treat the condition.X. Bayer’s experimental anticoagulant fails late-stage
trialOne of the
biggest disappointments from clinical trials came when a major late-stage trial for Bayer’s experimental anticoagulant asundexian had to be discontinued due to its inadequate effectiveness. Bayer had expectations in excess of € 5 billion (US$ 5.5 billion) from this drug.
Impressions: 2295
Endocrinology, a medicinal
branch that deals with endocrine and metabolic disorders, is witnessing
cutting-edge drug research to develop advanced drugs that can treat
diabetes, obesity, hypoparathyroidism and other
disorders.The endocrinology market is witnessing impressive growth — it is growing at a compound annual growth rate (CAGR) of 7.82 percent. Between 2023 and 2027, the market is poised to increase by US$ 36.5 billion. It includes treatments for various medical conditions, such as type 1 and type 2 diabetes, obesity, hypogonadism (a condition where the body’s sex glands produce little or no hormones), dwarfism, hypo or hyperparathyroidism, acromegaly (abnormal growth of the hands, feet, and face, caused by the overproduction of growth hormone by the pituitary gland) and congenital adrenal hyperplasia (a genetic disorder that affects the adrenal glands).The market is dominated by diabetes treatment drugs — valued at around US$ 48.7 billion in 2018, these are expected to generate US$ 78.3 billion by the end of 2026. The
market for obesity drugs is also expected to reach US$ 50 billion by 2030.The top companies in this
segment include Novo Nordisk, Eli Lilly, Merck, Sanofi, AstraZeneca and Boehringer Ingelheim. Several biotech companies
have joined the race to capture a share of the lucrative market for diabetes
and obesity drugs.Access the Endocrinology Newsmakers Dashboard (Free Excel)Lilly’s Mounjaro, Novo’s Wegovy gear up for larger share of obesity pieIn May 2022, Lilly’s potential blockbuster Mounjaro (tirzepatide) was approved by the US Food and Drug
Administration (FDA) as a once-weekly subcutaneous injection to treat adults with type 2 diabetes. Mounjaro is the first and only dual targeted glucose-dependent insulinotropic peptide (GIP) and glucagon-like peptide-1 (GLP-1) receptor agonist on the market, and has helped patients achieve a body weight loss of over 20 percent in clinical trials. Novo Nordisk’s Wegovy (semaglutide), the first and only once-weekly GLP-1 receptor agonist approved for weight management in people with obesity, has been found to help patients lose up to 15 percent of their body weight in over 68 weeks. In late 2022, the drug received FDA approval to treat obesity in pediatric patients aged 12 years and older.Last October, FDA granted fast track review designation to Mounjaro as a treatment for obesity. Lilly plans to go to the FDA soon and expects to get approval for the drug this year. Once approved, Mounjaro will give Wegovy a tough fight in the obesity market. Analysts predict Mounjaro to generate around US$ 8.1 billion in annual sales by 2028, while Wegovy is expected to bring in US$ 7 billion by 2030. Meanwhile, Amgen’s AMG 133 is also in early-stage trials for obesity. Access the Endocrinology Newsmakers Dashboard (Free Excel) ‘Skinny shot’ diabetes drugs face supply shortages; Lilly plans to double capacityFollowing its approval in the US in mid-2021, Wegovy quickly became the go-to “skinny shot” drug in Hollywood, thanks to its
impressive weight reduction data. But the drug hit a supply shortage in late 2021 due to manufacturing issues, which the manufacturer managed to fix in the second half of 2022. Novo’s blockbuster type 2 diabetes drug Ozempic is also in limited supply,
but the drugmaker expects the availability to return to normal around
mid-March.The supply constraints of Wegovy and Ozempic, coupled with the craze around using diabetes drugs for weight loss, sparked interest in Lilly’s Mounjaro. But Mounjaro, along with another Lilly drug Trulicity, began facing supply issues last December. While Lilly has fixed Mounjaro’s supply issues for the time being, it plans to double its manufacturing capacity by the year end to avoid any such shortages in the future.Meanwhile, the American Diabetes Association and Mutual Aid Diabetes have sounded an alarm, saying the off-label use of diabetes drugs for weight loss may cause shortages and affect diabetes patients.Access the Endocrinology Newsmakers Dashboard (Free Excel)Provention launches treatment for type 1 diabetes; Novo retains market dominanceIn November, Provention Bio’s Tzield (teplizumab) bagged FDA approval as a preventive treatment for type 1 diabetes in individuals eight years or older who are in stage 2 of the disease. The injectable drug is the first treatment in the US to delay progression of the autoimmune disease.Novo remained the dominant player in the diabetes and obesity market, clocking sales of US$ 22.8 billion (DKK 156.4 billion) in 2022 in this segment. Ozempic’s sales increased by 77 percent to US$ 8.6 billion (DKK 59.8 billion). The Danish company consolidated its position further by
purchasing Dicerna Pharma for US$ 3.3 billion in 2021 for its RNA interference (RNAi) technology.Novo is planning to file for approval of its once-weekly insulin icodec for type 2 diabetes in the US, the EU and China in the first half of 2023. It is also running a phase 1 trial of Ozempic as a once-weekly oral treatment.In 2022, sales of Lilly’s blockbuster drug Trulicity (dulaglutide) touched US$ 7.4 billion. Two other drugs, Humalog and Jardiance, generated US$ 2.1 billion each in sales. In addition, Lilly’s partner Boehringer Ingelheim also reported US$ 2.55 billion (€ 2.5 billion) sales of Jardiance in the first half of 2022.Biosimilar drugmaker Biocon Biologics acquired Viatris’ biosimilars business for a total of US$ 3.34 billion, strengthening its presence in the diabetes, tumors and autoimmune diseases market. Viatris’ portfolio is expected to generate sales of over US$ 1 billion for Biocon in 2023.Access the Endocrinology Newsmakers Dashboard (Free Excel)Drugmakers work on triple agonist therapies to
treat diabetes, obesitySeveral drugmakers are working on triple agonist therapies that target multiple receptors, such as GLP-1, GIP and glucagon (GCG). These drugs, such as Novo and partner Marcadia’s MAR423, Hanmi’s HM1521, Sanofi’s SAR441255, and Eli Lilly’s retatrutide, are currently in early- to mid-stage trials. Studies have shown that these triple agonists are more effective in reducing body
weight and improving glucose control compared to mono- or
dual-incretin receptor agonists.In addition, a few triple drug combinations containing metformin, DPP4 inhibitors and SGLT2 inhibitors have recently been approved. Treatments such as Astra’s Qternmet XR and Lilly-BI’s Trijardy XR have shown
significant improvement in glycemic control and are generally well tolerated by
patients.Access the Endocrinology Newsmakers Dashboard (Free Excel)TheracosBio’s oral med for diabetes approved; gene therapies gather interest Recently, TheracosBio’s Brenzavvy was approved by the agency as an oral
medication for type 2 diabetes. And Marinus Pharmaceuticals’ oral testosterone replacement therapy Kyzatrex received FDA’s nod last year for a range of diseases in men that arise from deficiency or absence of endogenous testosterone. New York-based Oramed is currently conducting a phase 1 trial of its oral GLP-1 drug – ORMD-0901 – as a treatment for type 2 diabetes.Meanwhile, Vertex and Arbor Biotechnologies are working on a gene therapy
(VX-880) for type 1 diabetes. BridgeBio Pharma’s BBP-631 adeno-associated virus (AAV) gene therapy for congenital adrenal
hyperplasia is in phase 1/2 trials. And
Swedish biotech Diamyd is carrying out phase 3 trials of its type 1 diabetes vaccine.In other medical conditions, Ascendis Pharma’s TransCon PTH, a long-acting prodrug of parathyroid hormone, has a PDUFA date in April
2023. If approved, it would become the first hormone replacement therapy to
address the underlying cause of hypoparathyroidism. Crinetics Pharmaceuticals’ Paltusotine, a non-peptide
somatostatin type 2 (SST2) receptor agonist, is currently in phase 3 trials for
acromegaly, representing a new class of oral once-daily medication.Access the Endocrinology Newsmakers Dashboard (Free Excel)Our viewDiabetes and obesity have
reached epidemic proportions in the recent decades, while other endocrine
disorders such as hypoparathyroidism are also growing rapidly the world over.According to the World Health Organization, around 422 million people worldwide have diabetes, and 1.5 million deaths are directly attributed to diabetes each year.While factors
such as long working hours, sedentary lifestyles, and unhealthy eating and
drinking habits are contributing to the rise of these diseases, drugmakers are
doing some cutting edge research for these conditions. Given this scenario,
endocrinology should see a lot of pathbreaking drugs in the coming years.
Impressions: 2157
The year 2022 saw the lowest number of drug approvals by the US Food and Drug
Administration (FDA) since 2016. The agency’s Center for Drug Evaluation
and Research (CDER) approved only 37 new drugs, a decrease of 26
percent from the 50 meds that it had approved in 2021. Of these, 21 were approved in the second half of the year (H2). Eight biologics were approved by FDA’s Center for Biologics Evaluation and
Research (CBER) in 2022 (against 10 in 2021). The FDA cited the Covid-19 pandemic as one of the main reasons
behind the drop in drug approvals.
Among the promising drugs
approved in H1 2022 were Eli Lilly’s Mounjaro (tirzepatide) for type 2
diabetes, Alnylam Pharmaceuticals’ rare disease drug Amvuttra (vutrisiran), Roche’s bispecific antibody for eye – Vabysmo (faricimab) – and Janssen Biotech and Legend Biotech's CAR-T therapy — Carvykti — to treat multiple myeloma. The second half saw some equally promising drugs bag FDA nod, such as Mirati’s Krazati (adagrasib) and Johnson and Johnson’s Tecvayli (teclistamab-cqyv), along with three of the costliest gene
therapies.
In 2022, 44 drugs were authorized by the European Medicines Agency (EMA), five less than the 49 authorized in 2021. Health Canada approved 20 drugs in H2, taking the total approvals to 45 in 2022.
View New Drug Approvals in 2022 with Estimated Sales (Free Excel Available)
Mirati’s Krazati, J&J’s Tecvayli bag approval; may clock around US$ 2 billion in peak sales
Oncology drugs continued to account for the largest number of approvals in H2 – nearly 35 percent of the drugs approved by CDER and CBER were from the therapeutic area. In H2 2022, cancer drugmaker Mirati’s Krazati became the second small molecule (KRAS) inhibitor to receive FDA approval for the treatment of advanced lung cancer. Amgen’s Lumakras was the first KRAS inhibitor to be approved in the US in May 2021. Krazati is expected to generate US$ 2 billion in peak sales by 2028. J&J’s Tecvayli was approved by the FDA as a fifth-line therapy for multiple myeloma. The drug is expected to generate peak sales of around US$ 1.7 billion.
View New Drug Approvals in 2022 with Estimated Sales (Free Excel Available)
FDA okays Gilead’s HIV drug, BMS’ plaque psoriasis drug, Amylyx’s ALS drug
Gilead Sciences’ long-acting HIV drug
Sunlenca (lenacapavir) finally bagged an FDA approval in H2, after receiving a complete response letter (CRL) in H1. The European Commission also authorized the drug to treat
patients who suffer from multi-drug resistant HIV.
FDA has also approved
Bristol Myers Squibb’s oral drug Sotyktu (deucravacitinib) as a treatment for moderate-to-severe plaque psoriasis. It’s the only TYK2 inhibitor approved for this condition and the first oral treatment approved in almost 10 years.
One of the most notable drug approvals of 2022 was Amylyx Pharmaceuticals’ Relyvrio, a treatment for
amyotrophic lateral sclerosis (ALS).
During H2, Lilly’s Mounjaro, Alnylam’s Amvuttra and Roche’s Vabysmo also received marketing authorization in Europe. AstraZeneca’s Tezspire was authorized in the EU as a treatment for severe asthma.
View New Drug Approvals in 2022 with Estimated Sales (Free Excel Available)
CSL’s Hemgenix emerges as costliest med; Bavarian Nordic’s monkeypox jab bags EUA
The second half saw several gene therapies bag an FDA nod – such as CSL’s Hemgenix (for
hemophilia B) and bluebird bio’s Skysona and Zynteglo (beti-cel). Priced at US$ 3.5 million per dose, Hemgenix became the most expensive treatment in the world, snatching the title from Skysona (priced at US$ 3
million), which was approved for a rare neurological disorder – cerebral adrenoleukodystrophy (CALD). Zynteglo (to treat beta thalassemia) was approved a month prior to Skysona, and is the third most expensive drug in the US (priced at US$ 2.8 million). Ferring’s bladder cancer gene
therapy, Adstiladrin, also received a regulatory nod, after getting rejected by
the FDA in May 2020.
With a rise in monkeypox
cases, the World Health Organization declared monkeypox as a global health emergency in July. The European
Commission gave marketing authorization to Bavarian Nordic’s Imvanex vaccine as a protection against monkeypox. The FDA granted emergency use authorization (EUA) to the same vaccine – branded as Jynneos – in August.
View New Drug Approvals in 2022 with Estimated Sales (Free Excel Available)
FDA rejects Y-mAbs’ cancer drug Omblastys, Gilead’s hepatitis med bulevirtide
One of the most noteworthy drugs that received FDA’s CRL is Y-mAbs Therapeutics’ Omblastys. The drug, meant to treat central nervous system metastasis from neuroblastoma, also received a negative recommendation from the EMA.
FDA also issued a CRL to Gilead Sciences’ hepatitis delta virus drug bulevirtide, citing concerns over the
manufacturing and delivery of the drug. The agency also rejected Ipsen’s palovarotene, an experimental
drug to treat fibrodysplasia ossificans progressiva (a tissue disorder),
requesting additional trial information.
View New Drug Approvals in 2022 with Estimated Sales (Free Excel Available)
Our view
Though 2022 was a lackluster year for drug approvals, 2023 has got off to a good start.
In the first week, Eisai and Biogen received FDA’s accelerated approval for their second Alzheimer's disease drug – Leqembi (lecanemab) – to treat patients who are in their earliest stages of the disease. Another Alzheimer’s drug – Eli Lilly’s donanemab – is up for review in February.
The year should also see the approval of the
first respiratory syncytial virus (RSV) vaccine in the US. GSK and Pfizer’s RSV vaccines for
older adults, along with Sanofi-AstraZeneca’s therapy for infants, are up for FDA review in 2023. Moderna also plans to seek approval for its RSV
vaccine for older adults in H1 this year. Moreover,
hemophilia A gene therapies from Sanofi (efmoroctocog alfa) and BioMarin (valoctocogene
roxaparvovec) are also up for
approval. All in all, this should be an exciting year for new drug approvals.
Impressions: 2357