Does Cytokinetics, Incorporated have a rare disease division?

Cytokinetics, Incorporated is a diversified pharmaceutical company headquartered in South San Francisco, United States. While it operates across multiple therapeutic areas, it maintains a significant rare disease portfolio with 4 active rare disease programs including Amyotrophic Lateral Sclerosis, Familial Hypertrophic Cardiomyopathy, Myasthenia Gravis. Many of its rare disease treatments hold orphan drug designations from the FDA.

How can I find Cytokinetics, Incorporated clinical trials for rare diseases?

The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Cytokinetics, Incorporated, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.

How do I enroll in a Cytokinetics, Incorporated clinical trial?

To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.

What FDA-approved rare disease drugs does Cytokinetics, Incorporated have?

Cytokinetics, Incorporated has 1 FDA-approved drug with a rare disease use that we track, as of October 2026. Myqorzo (aficamten), approved 2025: Familial Hypertrophic Cardiomyopathy. Source: the FDA's openFDA label database, matched to the rare diseases on Trial Friend; the list updates automatically and can include older products.

Does Cytokinetics, Incorporated offer copay help or free medicine for its rare disease drugs?

Yes, for 1 of its FDA-approved rare disease medicine that we track, each checked on Cytokinetics, Incorporated's own website. Myqorzo: MYQORZO & You (Cytokinetics), 833-697-6796. Offers copay help with private insurance, a free medicine program, a short-term supply while insurance is decided and insurance help. Checked September 29, 2026. Medicare and Medicaid: drugmaker copay cards are limited to private insurance, so people with government coverage usually look to a free medicine program or a charity fund, both on the help paying page. Eligibility is decided by each program, so call the number listed before a prescription is filled.

Cytokinetics, Incorporated

Q: What rare diseases does Cytokinetics, Incorporated focus on?

Cytokinetics, Incorporated has active programs across 4 rare diseases: Amyotrophic Lateral Sclerosis, Familial Hypertrophic Cardiomyopathy, Myasthenia Gravis, Spinal Muscular Atrophy. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.

Drugmaker with rare disease work · based in South San Francisco, United States · ticker CYTK · cytokinetics.com

Cytokinetics, Incorporated works on 4 rare diseases we track, including Amyotrophic Lateral Sclerosis, Familial Hypertrophic Cardiomyopathy and 2 more. It has 4 active clinical trials, and 2 are looking for volunteers now. It also has 1 FDA-approved rare disease drug.

Cytokinetics is a biopharmaceutical company in South San Francisco, California, that develops medicines for diseases of heart muscle function. Its drug Myqorzo (aficamten) is approved in the US for adults with symptomatic obstructive hypertrophic cardiomyopathy, and the FDA is reviewing a label update based on the MAPLE-HCM trial, with a decision due November 14, 2026. The company has also run trials in ALS, spinal muscular atrophy and myasthenia gravis.

Cytokinetics, Incorporated pipeline

Each row lists the diseases Cytokinetics, Incorporated is studying at that stage, from the ones closest to approval down to the earliest work.

Approved

Drugs the FDA has approved.

Myqorzo (aficamten)

Phase 3

Large studies, usually the last step before a company asks the FDA for approval.

Familial Hypertrophic Cardiomyopathy 2 trials

After approval

Studies that follow people already taking an approved drug.

Familial Hypertrophic Cardiomyopathy recruiting

Other studies

Studies with no phase listed, such as registries and natural history studies.

Familial Hypertrophic Cardiomyopathy recruiting

Cytokinetics, Incorporated clinical trials

These are Cytokinetics, Incorporated trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.

2 trials looking for volunteers now

Looking for volunteersRecently updatedNCT07054073

HCMR Re-Imaging Study

Tests None - observational study

For Hypertrophic Cardiomyopathy (HCM)

This study aims to learn what might predict heart problems (like sudden death from a fast heart rhythm or heart failure) in people with a genetic condition called hypertrophic cardiomyopathy (HCM). HCM causes the heart muscle to become thick, which can make the heart stiff and harder to work properly. It can also affect the heart's electrical system. This study is looking to enroll patients that were previously part of a research project called "HCMR - Novel Predictors of Outcome in Hypertrophic Cardiomyopathy." The results of that study are still being reviewed, but they might show that people who had a substance called Gadolinium (MRI contrast or dye) collected in their heart muscle may have a higher risk for heart problems, including sudden cardiac death. This is called "late gadolinium enhancement" (LGE). This study is aiming to do follow-up imaging on those patients to better understand how LGE affects people with HCM.

Ages 18 Years - 65 Years8 locations

View full study

Looking for volunteersPhase 4Updated a few months agoNCT07600177

Mavacamten to Aficamten Transition in Patients With Obstructive Hypertrophic Cardiomyopathy

Tests Aficamten

For Hypertrophic Cardiomyopathy (HCM)

This is an investigator-initiated two-center study. The goal of this study is to investigate the feasibility, safety and efficacy outcomes of a seamless transition from mavacamten to aficamten in patients with obstructive hypertrophic cardiomyopathy (oHCM).

Ages 18 Years - 90 Years2 locations

View full study

2 trials underway, not taking new volunteers

Underway, not taking new volunteersPhase 3Recently updatedNCT06081894

A Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM)

Tests Aficamten, Placebo

For Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy

This clinical trial will study the effects of aficamten (versus placebo) on the quality of life, exercise capacity, and clinical outcomes of patients with non-obstructive hypertrophic cardiomyopathy.

Ages 18 Years - 85 Years180 locations

View full study

Underway, not taking new volunteersPhase 2/3Recently updatedNCT06412666

A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM).

Tests Aficamten, Placebo

For Pediatric and Symptomatic Obstructive Hypertrophic Cardiomyopathy

The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of aficamten in a pediatric population with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).

Ages 12 Years - 17 Years36 locations

View full study

Ask about Cytokinetics, Incorporated's trials

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FDA-approved drugs from Cytokinetics, Incorporated

From the openFDA database. Tap a drug for side effects, dosing and help paying.

Myqorzo aficamten

For Familial Hypertrophic Cardiomyopathy. Approved Dec 19, 2025. Taken by mouth.

More about Cytokinetics, Incorporated

Within its broader pharmaceutical portfolio, Cytokinetics, Incorporated has active clinical trial programs and drug development efforts across 4 rare diseases, including Amyotrophic Lateral Sclerosis, Familial Hypertrophic Cardiomyopathy, Myasthenia Gravis, Spinal Muscular Atrophy. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.

The clinical trials section below shows active and recruiting rare disease studies sponsored by Cytokinetics, Incorporated, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites.

The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.

Cytokinetics, Incorporated is headquartered in South San Francisco, United States, publicly traded under the ticker symbol CYTK. The company maintains a dedicated rare disease division alongside its broader therapeutic portfolio, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.

Where Cytokinetics, Incorporated trials take place

The trials above run at 226 study sites in 20 countries. Tap a country to see its states, cities and hospitals.

United States

92▼

Japan

27▼

Spain

15▼

United Kingdom

12▼

China

12▼

Italy

11▼

Brazil

9▼

France

8▼

Canada

6▼

Israel

5▼

Portugal

5▼

Germany

4▼

Rare diseases Cytokinetics, Incorporated works on

Each disease page lists every open trial, not only Cytokinetics, Incorporated's, plus approved treatments.

Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis is a progressive neurodegenerative disease destroying motor neurons in the brain and spinal cord.

How common: About 5,000 new cases per year in the U.S.; CDC's National ALS Registry estimates about 33,000 Americans were living with ALS in 2022, a number projected to pass 36,000 by 2030

Familial Hypertrophic Cardiomyopathy

Familial hypertrophic cardiomyopathy is a genetic heart muscle disease characterized by inappropriate left ventricular hypertrophy and diastolic dysfunction.

How common: 1 in 500 people; 50% of first-degree relatives of affected individuals inherit the mutation

Myasthenia Gravis

Myasthenia gravis is a chronic autoimmune neuromuscular disorder causing muscle weakness and rapid fatigue.

How common: 1 in 5,000 people

Spinal Muscular Atrophy

Spinal muscular atrophy is an autosomal recessive genetic disorder affecting motor neurons in the spinal cord, causing progressive muscle weakness and atrophy.

How common: 1 in 10,000 to 14,000 births; 1 in 25 people are carriers

Where to get support

Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Cytokinetics, Incorporated's own site is cytokinetics.com, and our help paying for treatment page lists drugmaker programs and charity funds.

NIH GARD - Familial Hypertrophic Cardiomyopathy
Familial Hypertrophic Cardiomyopathy and Myasthenia Gravis
The ALS Association
Amyotrophic Lateral Sclerosis
ALS Therapy Development Institute
Amyotrophic Lateral Sclerosis
Hypertrophic Cardiomyopathy Association
Familial Hypertrophic Cardiomyopathy
Myasthenia Gravis Foundation of America (MGFA)
Myasthenia Gravis
SMA Foundation
Spinal Muscular Atrophy
Cure SMA
Spinal Muscular Atrophy

Cytokinetics, Incorporated

Cytokinetics, Incorporated pipeline

Cytokinetics, Incorporated clinical trials

2 trials looking for volunteers now

HCMR Re-Imaging Study

Mavacamten to Aficamten Transition in Patients With Obstructive Hypertrophic Cardiomyopathy

2 trials underway, not taking new volunteers

A Trial to Evaluate the Efficacy and Safety of Aficamten Compared to Placebo in Adults With Symptomatic Non-Obstructive Hypertrophic Cardiomyopathy (nHCM)

A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM).

FDA-approved drugs from Cytokinetics, Incorporated

More about Cytokinetics, Incorporated

Where Cytokinetics, Incorporated trials take place

Rare diseases Cytokinetics, Incorporated works on

Where to get support

Other companies working on the same diseases

Questions about Cytokinetics, Incorporated