By Aashi Mishra, Research Nester
Oligonucleotide APIs belong to a category of compounds that comprise short sequences of DNA or RNA, usually ranging from eight to 100 nucleotides. These compounds can be tailor-made to target an individual gene’s messenger RNAs (mRNAs) or other vital elements within cells. Oligonucleotide-based treatments are considered an advancement in genetic medicine, holding promise for tackling various illnesses such as genetic disorders, cancers, rare diseases, and infectious diseases.
The market for oligonucleotide APIs is projected to grow to $5 billion by 2035, with an annual growth rate of 10% from 2022 to 2035.
A significant trend in the market revolves around the rising need for personalized medicine, where targeted therapies are developed according to an individual patient’s traits. Another area experiencing growth is the utilization of APIs in gene therapy, which has demonstrated encouraging outcomes in addressing genetic disorders.
Driving Forces Behind The Demand For Oligonucleotide APIs
Increasing prevalence of genetic diseases: The number of genetic diseases is on the rise, impacting countless individuals worldwide. It is estimated that there are over 10,000 diseases caused by genetic mutations affecting more than 350 million people globally. This increasing prevalence of genetic diseases has resulted in a growing need for oligonucleotide API, which can be utilized to address various genetic disorders. Oligonucleotide therapies function by targeting genes or RNA molecules, allowing for precise treatment of genetic diseases. With research into and the development of new therapies, the demand for oligonucleotide API is expected to continue increasing.
Advancements in oligonucleotide synthesis technology: The progress made in oligonucleotide synthesis technology has brought about changes in the production of oligonucleotide APIs. These improvements have resulted in enhanced effectiveness, cost reduction, and better-quality management during manufacturing. A notable breakthrough in this field is the utilization of microfluidic systems, which provide accurate control over reaction conditions and enable the synthesis of high-grade oligonucleotides on a larger scale compared to previous capabilities.
Growing interest in personalized medicine: With the advancement of personalized medicine there has been a growing demand for therapies that can be customized to an individual’s unique genetic composition. This is where oligonucleotide API comes into play. By targeting certain genes or proteins, oligonucleotide API holds immense potential for transforming our approach to treating various diseases. For instance, oligonucleotide API has already demonstrated its effectiveness in managing spinal muscular atrophy (SMA), a rare genetic disorder impacting muscle strength and movement. By addressing the root cause of the illness, oligonucleotide therapy has exhibited promising results in improving motor function and extending survival rates among SMA patients.
Regulatory support: The regulations in place have an impact on how the pharmaceutical industry, including the market for oligonucleotide APIs, grows and develops. At times, these regulations have played a crucial role in increasing the demand for oligonucleotide APIs.
- One notable example is the FDA approval of Alnylam Pharmaceuticals’ Onpattro (patisiran), an RNAi therapeutic, in 2018. This approval marks a milestone as it is the first-ever treatment for the polyneuropathy that arises from hereditary transthyretin-mediated amyloidosis in adults.
- Another notable example is that the European Medicines Agency (EMA) approved Spinraza (nusinersen), an antisense oligonucleotide, to be marketed in the EU in 2017. This medication is intended for the treatment of spinal muscular atrophy (SMA), a genetic disorder that is both rare and potentially life-threatening.
These regulatory approvals have paved a path for advancing and commercializing therapeutics based on oligonucleotides. As a result, the field is attracting investments and fostering innovation.
Types Of Oligonucleotide APIs
- short interfering RNA (siRNA)
- antisense oligonucleotides
- phosphorodiamidate morpholino oligonucleotides (PMO)
- CpG oligonucleotides
- miRNA API and miRNA inhibitors
Each has its unique applications and therapeutic potential. siRNAs are utilized for reducing the expression of specific genes, while antisense oligonucleotides are employed for increasing the expression of specific genes. PMOs and aptamers are used for gene targeting and CpG oligos activate the immune system. miRNA APIs are used for gene targeting, and miRNA inhibitors are used for gene silencing.
Among all these approaches, it is anticipated that antisense oligonucleotides will capture the market share in the field of oligonucleotide APIs by 2035. Antisense oligonucleotides hold potential in addressing the root causes of Alzheimer’s disease rather than just alleviating its symptoms. Encouraging outcomes from trials have already been observed, making them an appealing choice for pharmaceutical companies and likely to gain popularity in the years ahead.
Application: Oligonucleotide APIs find applications in various fields, including genetic disorders, cancer, infectious diseases, and rare diseases. Among these, using oligonucleotides in cancer treatment is becoming more popular because they can deliver agents directly to cancer cells, causing minimal harm to healthy cells. Moreover, oligonucleotides have the potential to stop the production of proteins, which can effectively hinder tumor growth.
While the oligonucleotide API market holds immense promise, it also faces certain challenges:
- Cost: Creating and producing drugs that are based on oligonucleotides can be an expensive process, which could potentially restrict patients’ ability to access them.
- Regulatory Hurdles: Stringent regulations, especially when it comes to gene therapy, have the potential to impede the approval process and drive up the costs of development.
- Intellectual Property Issues: The oligonucleotide-based therapies field is filled with competition as companies vigorously safeguard their intellectual property rights, which could potentially result in legal conflicts.
Major Players And Recent Developments
The leading players in the market are Ionis Pharmaceuticals, Jazz Pharmaceuticals, Biogen, Sarepta Therapeutics, Alnylam Pharmaceuticals, Corden Pharma International, Nitto Denko Avecia, Merck KGaA, Ajinomoto Co., and Kaneka Eurogentec, among others. Recent developments are:
- On Oct. 31, 2022, EUROAPI revealed its plan to invest €18 million in upgrading its manufacturing equipment at its Frankfurt facility. This strategic move aims to cater to the increasing demand for peptides and oligonucleotides from CDMOs.
- On March 28, 2022, Ionis Pharmaceuticals, in partnership with Biogen Inc., shared the findings of their study on the application of antisense oligonucleotide (ASO) for individuals suffering from lateral sclerosis (ALS) associated with C9orf72.
- On December 22, 2021, Novartis’ Leqvio (inclisiran), a siRNA, received FDA approval. This innovative treatment enables cholesterol reduction and maintenance with two doses per year.
- On June 15, 2020, Jazz Pharmaceuticals revealed that the FDA had given its approval for Zepzelca (lurbinectedin) to be used in the treatment of adult patients who have metastatic cell lung cancer (SCLC) and have experienced disease progression following platinum-based chemotherapy.
Currently, there are 130 clinical trials for these oligonucleotides involving 80 different compounds. These trials cover a range of indications across 14 therapeutic areas and target 66 different genes.
The North American market for oligonucleotide API is expected to have the highest market share compared to other regions by the end of 2035. This growth can be mainly attributed to the rise in healthcare expenditures and improvements in healthcare facilities. According to a report by the Centers for Medicare & Medicaid Services, healthcare services in the United States accounted for approximately 19.7% of the country’s GDP in 2020, totaling $4.1 trillion. The rise in healthcare spending has resulted in advancements in facilities, including better access to new treatments and enhanced medical equipment.
It is projected that the European market for oligonucleotide APIs will witness substantial growth by the end of 2035. This growth can primarily be attributed to the escalating investments in research and development activities within the region, coupled with the presence of leading industry players. For instance, the biopharmaceutical sector in Europe witnessed approximately $21 billion in investments in 2018, followed by a surge of nearly 26% to reach $28 billion in 2019. Additionally, there is a growing awareness among patients regarding treatments and an increased focus on healthcare infrastructure development by governments, which is anticipated to fuel sector growth across Europe.
The oligonucleotide API market is bringing about a transformation in genetic medicine, with the potential to address various diseases by targeting their genetic origins. Although there are obstacles to overcome, this market is expected to experience growth due to technological advancements, broader therapeutic applications, and increased investment. As research and development efforts progress, it is highly likely that treatments based on oligonucleotides will play a crucial role in shaping the future of healthcare. This will pave the way for precise, effective, and personalized treatments for patients worldwide.
About The Author:
Aashi Mishra is a content developer at Research Nester and is also an electronics engineer. She has three years of market research experience.