Signifor (pasireotide)
Also marketed as Signifor LAR. An approved treatment for Cushing Disease.
The same compound appears under different names depending on the context. Here is how to identify Pasireotide wherever you encounter it, plus the key facts at a glance.
- Generic name
- Pasireotide
- Brand names
- Signifor, Signifor LAR
- Development code
- SOM230
- Drug class
- Somatostatin analog (pituitary-targeted)
- Manufacturer
- Recordati Rare Diseases
- How it's taken
- Two formulations: Signifor is given as a subcutaneous injection twice daily (starting dose 600 or 900 mcg).
The only pituitary-targeted medical therapy for Cushing's disease. While other medications work at the adrenal gland or receptor level, pasireotide acts directly on the ACTH-secreting pituitary tumor to reduce the signal that drives excess cortisol production. Available as twice-daily subcutaneous injection (Signifor) or monthly intramuscular injection (Signifor LAR).
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Where Pasireotide fits
The only medical therapy that targets the pituitary tumor directly. Approved for adults with Cushing's disease (pituitary-dependent) who cannot have pituitary surgery or for whom surgery did not cure the disease. Hyperglycemia risk is a major consideration in treatment selection.
How Pasireotide works
In Cushing's disease, a benign pituitary tumor produces too much ACTH, which tells the adrenal glands to make excess cortisol. Pasireotide binds to somatostatin receptors (especially SSTR5, which is highly expressed on corticotroph tumor cells) on the surface of these tumor cells, suppressing ACTH secretion at the source. This is the only approved medical therapy that targets the underlying pituitary tumor rather than blocking cortisol downstream.
Mechanism: Multi-receptor somatostatin analog that targets ACTH-secreting pituitary tumor cells to reduce ACTH and cortisol production
Side effects and safety
Hyperglycemia (high blood sugar) is the most significant side effect, occurring in about 73% of patients in clinical trials. Some develop new-onset diabetes requiring glucose-lowering medication. Other common effects in the main Signifor trial were diarrhea (58%), nausea (52%), gallstones (30%), headache (28%), and abdominal pain (24%). Blood sugar monitoring is essential. Because pasireotide lowers cortisol, cortisol can drop too low (hypocortisolism), causing weakness, tiredness, loss of appetite, nausea, vomiting or low blood pressure; tell your doctor right away if these happen. Pasireotide can also slow the heart rate and cause a heart rhythm change called QT prolongation, so an ECG is done before and during treatment, and it can raise liver enzymes, so liver tests are checked regularly. The label also warns about gallstone complications, drops in other pituitary hormones, loose or fatty stools from poor fat absorption, and very high blood sugar that can lead to a dangerous acid buildup called ketoacidosis.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Pasireotide
Two formulations: Signifor is given as a subcutaneous injection twice daily (starting dose 600 or 900 mcg). Signifor LAR is given as a monthly intramuscular injection (10 mg, 20 mg, 30 mg, or 40 mg) administered by a healthcare professional. The LAR formulation is generally preferred for convenience.
Availability and cost
Only available as the brand-name product.
Multi-receptor somatostatin analog available in two formulations (daily subcutaneous or monthly intramuscular). The only pituitary-targeted medical therapy for Cushing's disease.
Help paying for Signifor
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Copay help
Copay assistance is available to reduce out-of-pocket costs for eligible patients. Recordati states a $20 co-pay for eligible commercially insured patients.
For: private insurance · source - Other support
R.A.R.E. coordinates patient assistance programs for eligible patients who are uninsured or underinsured. Restrictions apply.
For: no insurance, underinsured · source - Insurance and case manager help
Help with insurance verification, prior authorization, specialty pharmacy delivery, and a clinical nurse specialist for medication questions.
The official page does not say who qualifies. Ask the program. · source
Good to know: SIGNIFOR and SIGNIFOR LAR are marketed in the US by Recordati Rare Diseases, not Novartis. The Cushing's page does not state the copay dollar amount; the $20 figure comes from the acromegaly patient page for the same program. Free-drug income rules are not published.
- From a charity · HealthWell FoundationCushing's Disease or Cushing's Syndrome fundOpen
Pays for: Copays, premiums or other treatment costs.
- From a charity · NORD RareCareCushing's Syndrome Medical Assistance fundOpen
Pays for: Medical and medication costs.
The foundation says: “Accepting new applications and re-enrollments for current year” - From a charity · NORD RareCareCushing's Syndrome Premium Copay Assistance fundOpen
Pays for: Insurance premiums and copays.
The foundation says: “Accepting new applications and re-enrollments for current year” - From a charity · TotalAssist (formerly PAN Foundation)Cushing's Disease or Syndrome fundOpen
Pays for: Out-of-pocket costs for approved medications, up to $9,500 per year. Requires health insurance (any kind).
How Cushing disease treatments compare
Four FDA-approved medical therapies are available for Cushing's disease. Your endocrinologist will recommend a treatment based on the source of your excess cortisol, your blood sugar levels, surgical history, and how you respond to initial therapy. This chart summarizes the key practical differences between options.
4 FDA-approved cushing disease treatments are available: Isturisa (osilodrostat, approved 2020); Korlym (mifepristone, approved 2012); Recorlev (levoketoconazole, approved 2021); Signifor LAR (pasireotide, approved 2012). Isturisa is typically used as first-line medical therapy when surgery is not possible or has not been curative.
| Drug | How it works | How it’s given | How often | Where you get it | Typical use | FDA approved |
|---|---|---|---|---|---|---|
Isturisa osilodrostat | Cortisol synthesis inhibitor — blocks 11-beta-hydroxylase, the final enzyme step in cortisol production | Oral tablet | Twice daily | Home (self-administered) | First-line medical therapy when surgery is not possible or has not been curative | 2020 |
Korlym mifepristone | Glucocorticoid receptor blocker — blocks cortisol from binding to its receptor (does not lower cortisol levels) | Oral tablet | Once daily with a meal | Home (self-administered) | For Cushing's patients with type 2 diabetes or glucose intolerance when surgery is not an option or has failed | 2012 |
Recorlev levoketoconazole | Cortisol synthesis inhibitor — blocks multiple steroidogenic enzymes including 11-beta-hydroxylase | Oral capsule | Twice daily with food | Home (self-administered) | For adults with Cushing's syndrome when surgery is not possible or has not been curative | 2021 |
Signifor LAR pasireotide You are here | Pituitary-targeted somatostatin analog — suppresses ACTH secretion from the tumor, reducing the signal that drives cortisol production | Intramuscular injection (monthly) or subcutaneous injection (twice daily) | Monthly (LAR) or twice daily (subcutaneous) | LAR: doctor's office or infusion center; Subcutaneous: self-administered at home | For Cushing's disease (pituitary-dependent) when surgery is not an option or has failed; only drug that targets the tumor itself | 2012 |
This comparison is for FDA-approved medical therapies only and does not include surgical or radiation treatments, which are typically first-line for Cushing's disease. Always discuss treatment selection with your endocrinologist, who can factor in tumor size, cortisol severity, metabolic complications, and your individual response.
Clinical trial results
Phase 3 trial: at 6 months, 26% of patients on 900 mcg and 15% on 600 mcg achieved normal urinary free cortisol. Signifor LAR approved in 2018 based on studies showing comparable efficacy with monthly dosing. Long-term extension data showed sustained cortisol reduction.
Development history
Signifor (subcutaneous) was the first FDA-approved medication for Cushing's disease in December 2012. Signifor LAR (monthly injection) was approved in 2018, offering a more convenient dosing schedule. Both formulations were developed by Novartis and are now sold in the US by Recordati Rare Diseases.
Explore Cushing Disease trials
Other Cushing Disease treatments
Common questions about Pasireotide
▸What is Pasireotide (Signifor)?
The only pituitary-targeted medical therapy for Cushing's disease. While other medications work at the adrenal gland or receptor level, pasireotide acts directly on the ACTH-secreting pituitary tumor to reduce the signal that drives excess cortisol production. Available as twice-daily subcutaneous injection (Signifor) or monthly intramuscular injection (Signifor LAR).
▸How does Pasireotide work?
In Cushing's disease, a benign pituitary tumor produces too much ACTH, which tells the adrenal glands to make excess cortisol. Pasireotide binds to somatostatin receptors (especially SSTR5, which is highly expressed on corticotroph tumor cells) on the surface of these tumor cells, suppressing ACTH secretion at the source. This is the only approved medical therapy that targets the underlying pituitary tumor rather than blocking cortisol downstream.
▸What are the side effects of Pasireotide?
Hyperglycemia (high blood sugar) is the most significant side effect, occurring in about 73% of patients in clinical trials. Some develop new-onset diabetes requiring glucose-lowering medication. Other common effects in the main Signifor trial were diarrhea (58%), nausea (52%), gallstones (30%), headache (28%), and abdominal pain (24%). Blood sugar monitoring is essential. Because pasireotide lowers cortisol, cortisol can drop too low (hypocortisolism), causing weakness, tiredness, loss of appetite, nausea, vomiting or low blood pressure; tell your doctor right away if these happen. Pasireotide can also slow the heart rate and cause a heart rhythm change called QT prolongation, so an ECG is done before and during treatment, and it can raise liver enzymes, so liver tests are checked regularly. The label also warns about gallstone complications, drops in other pituitary hormones, loose or fatty stools from poor fat absorption, and very high blood sugar that can lead to a dangerous acid buildup called ketoacidosis.
▸How is Pasireotide taken?
Two formulations: Signifor is given as a subcutaneous injection twice daily (starting dose 600 or 900 mcg). Signifor LAR is given as a monthly intramuscular injection (10 mg, 20 mg, 30 mg, or 40 mg) administered by a healthcare professional. The LAR formulation is generally preferred for convenience.
▸Is Pasireotide FDA approved?
Yes, Pasireotide (Signifor) is FDA approved (2012) for the treatment of Cushing Disease.
▸How does pasireotide treat Cushing disease specifically?
In Cushing disease, a pituitary tumor produces excess ACTH, which drives the adrenal glands to overproduce cortisol. Pasireotide targets somatostatin receptors on the pituitary tumor cells, reducing ACTH secretion and consequently lowering cortisol levels.
▸Is this the same drug as pasireotide for acromegaly?
Yes, it is the same molecule (pasireotide/Signifor), but used for a different condition. The Cushing disease indication was approved first in 2012, while the acromegaly indication (Signifor LAR) was approved in 2014. The dosing and formulation may differ.
▸What is the main side effect concern?
Hyperglycemia (high blood sugar) is the most significant side effect, occurring very frequently due to pasireotide's effects on insulin secretion. Blood glucose monitoring is essential, and many patients require diabetes medications during treatment.
▸When is pasireotide used for Cushing disease?
Pasireotide is typically used when surgery on the pituitary tumor has not been curative or is not possible. It addresses the source of the problem (excess ACTH from the pituitary) rather than blocking cortisol production or action downstream.
▸How is Signifor administered for Cushing disease?
For Cushing disease, Signifor is available as a subcutaneous injection given twice daily. The long-acting Signifor LAR formulation (monthly intramuscular injection) is also used for some patients.
▸How does pasireotide compare to cortisol-lowering drugs?
Pasireotide targets the root cause (pituitary ACTH production), while osilodrostat and levoketoconazole block cortisol production in the adrenal glands, and mifepristone blocks cortisol at its receptor. The approach depends on the individual patient's disease characteristics.