Another Year of Blockbuster Drug and Treatment Approvals Possible in 2023
The United States Food and Drug Administration (FDA) canonical 45 new drugs in 2022.1
Though the number of approvals was the lowest since 2016, nearly half of them are expected to become blockbusters, meaning greater than $1 billion in annual sales.2
The approved cohort is impressive, with expectations especially loftier for Bristol Myers Squibb’s psoriasis therapy, Alnylam’s ATTR polyneuropathy drug, and Gilead’s new HIV treatment. The FDA likewise approved a tape five cell and gene therapies last year, bringing total canonical cell and gene therapies to 19.3, 4
Amongst them, bluebird bio’s beta thalassemia gene therapy and CLS-uniQure co-developed Hemgenix for Hemophilia B stand out.
In this piece, we look ahead to 2023 and what could be more heady developments in the biopharmaceuticals market. We highlight iii companies in the Global X Genomics & Biotechnology ETF (GNOM) and the Global X Crumbling Population ETF (AGNG) with noteworthy drugs upwards for approval.
- Eli Lilly: Extended approval for Mounjaro could pb to the most successful drug e’er.
- CRISPR Therapeutics: Forging a path with exa-cel, the commencement gene editing treatment.
- Sarepta Therapeutics: SRP-9001 may be the first cure for Duchenne muscular dystrophy.
Eli Lilly: Mounjaro Tin can Tap into High-Growth Obesity Marketplace
In 2022, the FDA and the European Medicines Agency (EMA) canonical Eli Lilly’s Mounjaro (Trizepatide) for Blazon ii diabetes. Eli Lilly’s endocrinology portfolio comprised an estimated 47% of its total revenue in 2022, led by blockbusters Trulicity and Jardiance.5
The two drugs brought in a combined $10 billion last year.six
With its adjacent-generation approval, Eli Lilly has a route towards long-term success in the $64 billion diabetes market.7
In 2023, Eli Lilly seeks expanded approving of Mounjaro for obesity, a motion that would nearly double the expected sales for the drug by 2028.8
Expanded approval would play a cardinal role in estimated almanac peak sales of $25 billion for Mounjaro, which would brand information technology the most successful drug ever, topping AbbVie’southward Humira, which achieved in an estimated $22 billion in 2022.9, 10
Diabetes & Obesity: Mounjaro Capitalizes on Converging Categories
Mounjaro is a dual-action drug that activates the GLP-one [glucagon-like peptide-one] and GIP [glucose-dependent insulinotropic polypeptide] receptors, both of which stimulate the body to produce more than insulin when a patient’south blood sugar levels get-go to rise. The handling is injected once a calendar week, with a dosage adjustable to the patient’s needs.
These drug categories also help adjourn hunger, opening its potential apply to the obesity market, which is expected to be worth over $50 billion by the end of the decade.11
The obesity market is expected to grow at a compound annual growth rate (CAGR) of 33% from $two.vii billion, propelled by the broader shift in healthcare to focus more on causes of illnesses like diabetes and cardiovascular disease rather than treatment of their symptoms.12
Prediabetes patients, for instance, could benefit from such treatments. Estimates prove that for every type 2 diabetic, a population of 30 meg patients in the United States, there are virtually twice equally many prediabetics.13, fourteen
Clinical Trial Data Suggest Mounjaro Tin Exist an Obesity Market Leader
In its SURMOUNT-1 clinical trial, Lilly reported that Mounjaro helped patients achieve average weight reductions of xvi%, 21.4%, and 22.5% at 5mg, 10mg, and 15mg doses, respectively.15
The results topped Novo Nordisk’s diabetes drug Ozempic, marketed as Wegovy to obesity patients, which in clinical trials helped patients lose an average 12.4% of their trunk weight versus placebo.sixteen
The medical community is excited about Mounjaro and what information technology could mean for the obesity market, as its efficacy data compares favorably to traditional surgical methods. Average weight reduction for sleeve gastrectomy surgery and gastric bypass are 20% and 25-thirty% respectively, only surgery-related risks are loftier, equally are their price tags at upwards of $35,000.17
CRISPR Therapeutics’ Exa-Cel Can Help Validate Gene Editing Market
Gene editing frontrunner CRISPR Therapeutics, in collaboration with Vertex Pharmaceuticals, is i step closer to bringing its investigational gene editing handling, exa-cel, to the market. This treatment allows scientists to edit parts of the genome by removing, adding, or altering sections of a patient’s Deoxyribonucleic acid sequence.
If approved, exa-cel’s sales are expected to height $1 billion in 2028, with expected market share of 20% in sickle cell and 36% in thalassemia.18, 19
An approval would validate the promise of gene editing techniques and open a market place estimated to be worth $5.seven billion in 2028 with far-reaching potential across a host of genetic disorders.20
In addition, the FDA’south review of exa-cel volition establish regulatory precedents and, if approved, pricing benchmarks for gene editing that will exist key in the field’s long-term success.
Exa-cel: A Curative Selection for Sickle Cell Disease and Beta-Thalassemia
Exa-cel is a potential 1-fourth dimension treatment for sickle cell disease and β-Thalassemia, two blood-based disorders that misconstrue the production and construction of hemoglobin, the protein in red blood cells that carries oxygen. Without enough normal hemoglobin, ruby-red blood cells alter shape, allowing them to clump together and block blood flow. Patients often require frequent transfusions to restore blood volume levels and oxygen period. Exa-cel edits the patient’due south stem cells to produce higher levels of hemoglobin, helping to alleviate or eliminate transfusion requirements for patients.
In its latest update, CRISPR and Vertex reported that 42 out of 44 participating patients with β-Thalassemia are transfusion free later on treatment, with follow-upward ranging from 1.2 to 37.2 months.21
This issue represents 95% efficacy, higher than the 89% for bluebird bio’southward Zynteglo, a one-time β-Thalassemia gene therapy approved past the FDA in August 2022.22
In sickle cell, all 31 patients treated with exa-cel were free of vaso-occlusive crises, with follow-up ranging from ii.0 to 32.3 months. On average, trial patients had hemoglobin levels of 43% 12 months subsequently infusion with stiff prophylactic data.23
Hemoglobinopathies Is an Increasingly Competitive Market
Hematological disorders have served every bit a proof of concept for new therapeutic modalities, such every bit gene therapy, cell therapy, and gene editing. A consequence is an increasingly competitive market for cumbersome claret-based genetic disorders similar sickle cell disease and β-Thalassemia. However, in our view exa-cel has a unique advantage in the market due to its reported clinical trial information and the expected timing of its market entry.
Bluebird bio, following FDA approval of its β-Thalassemia gene therapy, now seeks a green calorie-free for its sickle prison cell therapy in Q1 2023.24
Recent clinical trial data show that patients on the treatment had hemoglobin levels of 45% at 24 months after infusion.25
Notably, safety information might limit the appropriate puddle of patients for the therapy if approved. Gene therapy competitors Intellia and Editas are working on their own CRISPR-based therapies for sickle cell, though they are much earlier in their clinical development processes, giving CRISPR and Vertex an early pb.
Sarepta Therapeutics’ SRP-9001 Could Exist on the Approval Fast Rails
Sarepta Therapeutics, leader in the Duchenne musical dystrophy (DMD) handling space, looks to expand its footprint past offering a functional cure for the affliction via an investigational gene therapy. DMD is a rare disorder of progressive muscular weakness that occurs due to a mutation in the dystrophic gene. The disease usually affects boys in early childhood.
Sarepta has successfully commercialized three DMD therapies, each of which facilitates the creation of partially functional dystrophin via exon-skipping engineering science. In total, Sarepta’s exon-skipping therapies are a good lucifer for an estimated 29% of DMD patients.26, 27, 28
Sarepta’s combined DMD sales were expected to total $811 meg in 2022, representing the entire DMD treatment market. If Sarepta receives regulatory approval for its SRP-9001 therapy, its combined DMD-related sales could jump to $3.2 billion in DMD-related sales in 2028, led past SRP-9001’s $2 billion contribution.29
Sarepta expects total peak sales for SRP-9001 to attain $4 billion.xxx
SRP-9001 Changing Treatment Landscape for DMD
In Duchenne patients, a mutation in the DMD gene leads to a astringent lack of dystrophin production. Dystrophin is a protein that works to ensure the integrity and construction of muscle fibers in skeletal and heart muscles. Without dystrophin, normal activeness causes excessive damage to cells, and is replaced with fat and fibrotic tissue over time. Amongst symptoms, patients tin experience frequent falls, trouble getting up, difficulty running, and learning disabilities. On average, patients require a wheelchair by 10–12 years old and assisted ventilation by 20.31
Approximately one in every two,400 boys worldwide is born with DMD.32
Sarepta’south SRP-9001 is a cistron therapy designed to deliver dystrophin-encoding gene into a patient’southward muscle tissue to prompt production of dystrophin. The therapy shows strong improvement in clinical trial patients, with statistically meaning comeback in motor ability at 48 weeks later infusion.33
In its phase Ii trial, the twenty patients treated with the gene therapy had a hateful improvement of 1.3 points in the broadly used rating scale to measure functional motor abilities in children with DMD.34
In the same time frame, patients usually experience a 0.7-point decline in motor function.35
SRP-9001 Approval Can Requite Sarepta a Head Get-go in DMD Gene Therapy Market
If canonical on an accelerated timeline, SRP-9001 could be marketed right abroad, and Sarepta would submit information from its EMBARK clinical trial equally a mail-marketing study to confirm clinical benefit. The firm expects a decision by May 29, 2023.36
Approving by this engagement would give Sarepta meaning time equally the only such DMD therapy on the marketplace. The adjacent DMD therapy expected to come up to the market is Pfizer’s in 2024, at the earliest.37
Sarepta is as well expanding its inquiry to offer an unabridged portfolio of solutions for DMD. In collaboration with Harvard University and Duke Academy, it is investigating curative factor editing engineering to correct the DMD-causing mutation and produce fully functional dystrophin.
Conclusion: Pending Approvals Show Biotech’south Continued Promise
With potential approvals forthcoming, we believe Eli Lilly, CRISPR Therapeutics, and Sarepta Therapeutics tin can help pave the style towards a more effective patient treatment prototype beyond key disease areas. For investors, Mounjaro, exa-cel, and SRP-9001 represent latest round of breakthroughs in patient care, where the focus continues to shift from treating symptoms to addressing the root causes of diseases at the biological level. With exposure to the Genomics & Biotech and Aging Population investment themes, investors can capture breakthroughs like these and potentially benefit from their growth as they disrupt traditional patient care for the better over fourth dimension.
GNOM – Global Ten Genomics & Biotechnology ETF
AGNG – Global X Aging Population ETF
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