Advancing a strong and diverse pipeline

We are advancing more than 160 projects in full clinical development, with 54 ongoing Phase III programs. By 2026, we anticipate approval of 20 pipeline assets with the potential to become blockbuster medicines with annual sales of more than USD 1 billion. Building on our success in small-molecule therapies and biologics, we are also investing in advanced technologies that offer new treatment paradigms for patients, such as radioligand therapies and gene therapies.

Deliver transformative innovation – key figures1





Projects entering development pipeline2




Ongoing Phase III programs3




US FDA breakthrough therapy designations4




Major submissions (US, EU, JP, China)5




Major approvals (US, EU, JP, China)5




New molecular entity (NME) approvals6





Includes Innovative Medicines and Sandoz biosimilars only


Includes projects entering confirmatory development from internal R&D activities. First patient, first visit (FPFV) has occurred in post-proof-of-concept stage after NIBR


Includes projects with FPFV in a Phase III study but not yet filed in the US, the EU, Japan or China


Number of breakthrough therapy designations granted by the US Food and Drug Administration for therapies under development by Novartis


Includes small molecules, biologics; new fixed-dose combinations of existing APIs; and new target indications, defined as new disease or new line of treatment (e.g., first line vs. second line)


Includes NMEs such as small molecules, biologics; in the EU, new fixed-dose combinations of existing APIs

Cardiovascular, renal and metabolism

Cardiovascular disease (CVD) is the leading cause of death worldwide. It is one of several complex and often interrelated chronic disorders – along with renal and metabolic diseases – that together affect billions of people around the globe. Novartis is pioneering treatments that address the spectrum of these diseases.

Entresto is approved in more than 100 countries for the treatment of adult symptomatic heart failure with reduced ejection fraction, a condition in which the heart fails to pump blood as well as it should. In 2021, Entresto was granted an expanded indication in the US and other countries, allowing for the treatment of most chronic heart failure patients, including all those with an ejection fraction below normal.

Also in 2021, Entresto received approval in China and Japan for treatment of patients with essential hypertension, the most common form of high blood pressure. This new indication makes Entresto the first new therapy for hypertension in China in over 10 years.

Leqvio is a novel treatment that reduces low-density lipoprotein (LDL) cholesterol, a highly important modifiable risk factor for atherosclerotic cardiovascular disease (ASCVD), which accounts for over 85% of all CVD deaths. Clinical studies showed that this first and only small-interfering RNA therapy for ASCVD can reduce LDL cholesterol by up to 52%, on top of maximally tolerated statins, through two injections per year, after an initial dose and one at three months. Leqvio has been approved in more than 50 countries, including the US and the EU, as well as in the UK as part of a population health management agreement that is expected to reach up to 300 000 patients in three years.

TQJ230 (pelacarsen), another nucleic acid-based therapy, is currently in Phase III development for the secondary prevention of cardiovascular events in patients with elevated levels of lipoprotein(a), an independent inherited ASCVD risk factor that cannot be effectively addressed by diet and other lifestyle changes. Phase II trial data showed that TQJ230 can reduce lipoprotein(a) in high-risk patients below recognized risk thresholds.

LNP023 (iptacopan) is an investigational treatment for several severe, life-limiting kidney conditions, including C3 glomerulopathy (C3G) and IgA nephropathy (IgAN) – two diseases that mainly affect younger patients – as well as paroxysmal nocturnal hemoglobinuria, a life-threatening blood disorder. In 2021, we announced that LNP023 met its primary endpoints for C3G and its primary endpoint for IgAN in Phase II clinical trials. Phase III studies are ongoing.


Cancer, one of the world’s leading causes of death, inflicts a growing burden. Global cancer deaths are expected to nearly double by 2040 due mainly to population growth and aging.

Novartis is a leader in finding new treatments for cancer, with approximately 45 compounds in development across four therapeutic platforms: targeted treatments, radioligand therapies, cell therapies and immunotherapies.

Novartis is a leader in finding new treatments for cancer, with approximately 45 compounds in development

Lung cancer

More people die of lung cancer than any other type of cancer, with death rates increasing in many parts of the world.

ACZ885 (canakinumab) is an antibody treatment currently in Phase III development for non-small cell lung cancer. It was accelerated into lung cancer studies after results in a large-scale cardiovascular study demonstrated a significantly lower than expected lung cancer mortality. In 2021, we reported that two Phase III trials in non-small cell lung cancer for second- and first-line patients did not meet their primary endpoints. In the first-line study, however Novartis researchers observed potentially meaningful improvements among certain groups of patients. These results support continued study of ACZ885 in earlier stages of lung cancer and further evaluation of Pro-Tumor Inflammation in all lung cancer settings. Studies of ACZ885 in both the adjuvant and neoadjuvant setting are ongoing.

In our mid-stage pipeline, we are exploring the potential of two compounds discovered at NIBR – JDQ443 and TNO155 – to target the previously “undruggable” proteins KRAS and SHP2, respectively. If proven effective, the combination therapy could open up new treatment options for patients with lung cancer as well as other hard-to-treat tumors.

Our oncology pipeline also includes LXH254 (naporafenib), a targeted cancer therapy currently being studied in multiple combinations in defined populations of melanoma and lung cancer patients, and NIS793, for which the FDA has granted orphan drug designation for the treatment of pancreatic cancer in combination with chemotherapy.

Prostate cancer

Prostate cancer is the second most diagnosed cancer in people with a prostate gland, with poor survival prognosis in metastatic disease.

Novartis is exploring a new, targeted way to treat metastatic castration-resistant prostate cancer (mCRPC) with177Lu-PSMA-617 (lutetium Lu 177 vipivotide tetraxetan/lutetium (177Lu) vipivotide tetraxetan), an investigational radioligand therapy. In 2021, the FDA granted177Lu-PSMA-617 breakthrough therapy designation after a Phase III study showed that the treatment plus existing care options significantly improved overall survival and radiographic progression-free survival for patients with mCRPC compared to existing care options alone. Regulatory submissions for177Lu-PSMA-617 have been accepted by the FDA and European Medicines Agency.

Breast cancer

Breast cancer, the most common cancer in women, is responsible for more than 685 000 deaths per year.

We continue to see results from our clinical program for Kisqali. The results of a Phase III study announced in 2021 showed that Kisqali in combination with an aromatase inhibitor achieved an overall survival benefit of more than five years for postmenopausal women with hormone receptor-positive (HR+)/human epidermal growth factor receptor 2-negative (HER2-) advanced breast cancer, which is the most common subtype of the disease. The data represent the longest reported median survival from a randomized trial in HR+/HER2- advanced breast cancer. Novartis is also conducting a Phase III study of Kisqali with endocrine therapy in the adjuvant treatment of HR+/HER2- early breast cancer. The trial completed enrollment in 2021.

Piqray is approved in the US and the EU for breast cancer patients whose disease harbors a PIK3CA mutation. Results from an ongoing Phase II study presented in 2021, as well as recent guideline updates, highlight the efficacy of Piqray with fulvestrant for postmenopausal HR+/HER2-, PIK3CA-mutated advanced breast cancer patients immediately after failure on prior CDK4/6i treatment (a type of targeted treatment). Development is underway for five new indications, including PIK3CA-relative overgrowth syndrome, a group of rare disorders that cause overgrowth of parts of the body.


Scemblix, a new treatment for chronic myeloid leukemia (CML), was approved by the FDA in 2021 for use in patients who are resistant or intolerant to prior treatments, and also for patients with a specific mutation. Discovered by researchers at NIBR, the treatment highlights our ongoing commitment to patients with the disease (see “Two decades of pioneering innovation in chronic myeloid leukemia” below).

MBG453 (sabatolimab) is an anti-TIM-3 monoclonal antibody being studied for the treatment of higher-risk myelodysplastic syndromes and acute myeloid leukemia, both rare blood cancers. In 2021, MBG453 received a fast track designation from the FDA and an orphan drug designation from the European Commission. In 2021, we reported that a Phase III clinical study of Kymriah as a second-line treatment in aggressive B-cell non-Hodgkin lymphoma did not meet its primary endpoint. We continue to study Kymriah in other forms of lymphoma and leukemia.

In 2021, we reported that a Phase III clinical study of our chimeric antigen receptor T-cell (CAR-T) therapy Kymriah as a second-line treatment in aggressive B-cell non-Hodgkin lymphoma did not meet its primary endpoint. We continue to study Kymriah in other forms of lymphoma and leukemia.

Two decades of pioneering innovation in chronic myeloid leukemia (CML)

Scientists during the early days of research into targeted cancer therapies (Photo)

Photo Novartis scientists during the early days of research into targeted cancer therapies

With a breakthrough approval 20 years ago, Novartis opened the door to reimagine CML and other cancers. Despite these advancements, we’re not standing still.

In May 2001, Novartis received approval in the US for the first targeted therapy for cancer, known as a tyrosine kinase inhibitor. This was a watershed moment in drug discovery, transforming the treatment landscape for CML and opening the door to treatment possibilities for other forms of cancer and blood disorders. Today the estimated five-year survival rate for CML is above 70%; in the 1970s, it was only 22%.

Yet despite CML being transformed into a chronic disease for many patients, significant unmet needs still remain – particularly for patients who have experienced resistance or intolerance to available treatments. Our research continues, and in 2021 we received US approval of a new treatment for CML in two distinct indications, offering a new treatment option for patients who are resistant or intolerant to existing therapies, and marking another milestone in our long-standing scientific commitment to patients living with CML.

Immunology, hepatology and dermatology

Our R&D teams are pioneering the development of therapies for immune system disorders, skin conditions and other diseases with high unmet needs.

We are committed to developing medicines that will advance the treatment of chronic spontaneous urticaria (CSU), so patients are able to live their lives without the distressing and unpredictable symptoms of this skin disease. Any new therapies will add to our portfolio of medicines that already includes Xolair, our existing approved therapy for CSU.

Enrollment began in 2021 for Phase III studies in CSU of LOU064 (remibrutinib), an oral BTK inhibitor that we are investigating for a number of immune-mediated conditions.

Also in 2021, Phase III clinical trials for QGE031 (ligelizumab), a next-generation monoclonal anti-immunoglobulin E (IgE) antibody, showed that QGE031 demonstrated superiority compared with placebo in the treatment of CSU, but not versus Xolair. Novartis is continuing to evaluate the trial data. We also continue to evaluate the potential for QGE031 to bring benefit to patients in the areas of chronic inducible urticaria and food allergy, where there is significant unmet need.

Cosentyx, our treatment for several systemic inflammatory conditions, continues to show strength in addressing a variety of debilitating dermatological and rheumatological conditions. In 2021, we announced approvals of Cosentyx in the US and China for the treatment of moderate-to-severe plaque psoriasis in pediatric patients aged 6 years and older who are candidates for systemic therapy or phototherapy, as well as in the US to treat active juvenile psoriatic arthritis (JPsA) in patients aged 2 years and older, and active enthesitis-related arthritis (ERA) in patients aged 4 years and older. Cosentyx is the only biologic treatment approved for children and adolescents for both ERA and JPsA in the US.

Cosentyx is also being studied for other inflammatory conditions, including hidradenitis suppurativa, giant cell arteritis and lichen planus, highlighting our commitment to expand Cosentyx to more indications and provide new treatment options for patients.

In 2021, we announced the discontinuation of a Phase IIb study of CFZ533 (iscalimab) in kidney transplant patients. The study of CFZ533 in liver transplant continues, as do studies exploring CFZ533 as a potential treatment in other conditions, such as hidradenitis suppurativa and Sjögren’s syndrome.


Neurological diseases are a leading cause of death and disability worldwide. Our work in neurological disorders includes investigational treatments for multiple sclerosis (MS), spinal muscular atrophy (SMA) and other diseases.

Our gene therapy Zolgensma is a treatment for both presymptomatic and symptomatic children with SMA, a devastating neurodevelopmental disease. It is approved in 42 countries. Building on our success with Zolgensma, in 2021 we started a new Phase III study of an intrathecal formulation in treatment-naive patients with SMA type 2. Early patient identification through newborn screening is critical for SMA patients since the disease causes irreplaceable motor neuron loss. For more on how we work with payers and healthcare systems to highlight the importance of early screening for SMA patients, please see the section “Embrace operational excellence.”

Our comprehensive portfolio for MS – a debilitating chronic disease that affects 2.8 million people worldwide – emphasizes our commitment to improving the quality of life for people living with MS at all stages of the disease. Kesimpta, one of our therapies for MS, received approval in the EU in 2021 for the treatment of relapsing forms of MS in adults with active disease. It is already approved in the US and other key markets. Kesimpta is the first B-cell therapy that can be self-administered at home. In 2021, we also started Phase III trials to investigate LOU064 in patients with relapsing forms of MS.

Our portfolio shows our commitment to improving the quality of life for people living with multiple sclerosis

We are exploring other neurological conditions where the need for new treatments is pressing. One example is Huntington’s disease, a rare, inherited neurodegenerative condition that leads to progressive disability and death. There currently are no approved therapies that delay onset of the disease or slow its progression. LMI070 (branaplam), our investigational, oral small-molecule RNA splicing modulator, could potentially treat Huntington’s disease by targeting RNA to reduce levels of a mutant protein. Phase II trials were initiated in 2021.

In 2021, Novartis and UCB announced a global co-development and co-commercialization agreement to bring potentially disease-modifying therapies to people living with Parkinson’s disease, which affects more than 10 million people worldwide. These investigational therapies include UCB0599, a small-molecule, alpha-synuclein misfolding inhibitor currently in Phase II development, which is being studied to understand if it can slow or stop the progression of Parkinson’s disease. In addition, upon completion of an ongoing Phase I program, there is an opt-in to co-develop UCB7853, an anti-alpha-synuclein antibody.


We are working to find innovative treatments for diseases of the eye, with the goal of reducing or eliminating the huge burden on patients and society caused by visual impairment and blindness.

One of our novel treatments is UNR844, a topical ophthalmic solution currently in Phase II development for presbyopia – a common, gradual, age-related loss of the ability to focus actively on nearby objects, caused by loss of elasticity of the lens of the eye.

In 2021, Novartis announced an agreement to acquire the UK-based ocular gene therapy company Gyroscope Therapeutics, adding to our pipeline a one-time gene therapy that could transform care for geographic atrophy (GA), an advanced form of dry age-related macular degeneration (AMD) that leads to progressive and irreversible vision loss. There are no approved treatments for GA, making it one of the most significant unmet needs remaining in retinal diseases. GT005, the lead asset from Gyroscope Therapeutics, is designed as an AAV2-based, one-time investigational gene therapy for GA secondary to AMD that is delivered under the retina. Completion of the acquisition is subject to customary closing conditions. Novartis and Gyroscope Therapeutics will continue to operate as separate and independent companies until closing.

Respiratory and allergy

Novartis is developing novel therapies to treat respiratory conditions such as asthma, chronic obstructive pulmonary disease and fibrotic lung diseases. These illnesses affect the lives of millions of people worldwide, and evidence suggests the burden is increasing due to climate change.

Building on our success with Xolair, the first biologic brought to market for asthma, we are pursuing Phase II studies for CSJ117, an inhaled biologic adjuvant therapy for severe uncontrolled asthma. We also initiated Phase III studies of QGE031 for patients with IgE-mediated peanut allergy.