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Certain von Hippel-Lindau (VHL) disease-related tumors

Clinical trial results for WELIREG

How WELIREG was studied

Doctors conducted a clinical study with 61 adults taking three 40-mg pills of WELIREG once a day in order to measure the percentage of patients whose tumors shrank. A closer look at this study is provided below.

Patients

A clinical study included 61 adults with at least 1 VHL disease-related tumor in their kidneys. Of those, 24 also had VHL disease-related tumors in their brain and spinal cord and 12 had tumors in their pancreas.

Treatment

People in the study took three 40-mg pills (120 mg) once a day until the study provider determined it was appropriate to stop treatment due to disease worsening or if they had certain side effects.

Outcomes

The study measured the percentage of patients whose tumors shrank after starting treatment. The study also measured duration of response, which measured how long a patient responded to treatment, and time to response.

WELIREG helped certain VHL disease-related tumors shrink

Kidney (renal cell carcinoma)

At the time of patient follow-up:

49% of patients (30 out of 61) saw tumors in their kidneys partially shrink.

  • Patients who responded to treatment continued to respond from 2.8+ to 22+ months (median not reached).

Half of the patients whose kidney tumors responded to WELIREG had a response at 8 months or shorter. The range for time to response was from 2.7 to 19 months.

56% of these patients (17 out of 30) were still responding to treatment for 12 months or more.

Brain and spinal cord (central nervous system hemangioblastomas)

At the time of patient follow-up:

63% of patients (15 out of 24) saw tumors in their brain and spinal cord partially or completely shrink. 14 out of these 24 patients saw their tumors partially shrink and 1 saw their tumors completely shrink.

  • Patients who responded to treatment continued to respond from 3.7+ to 22+ months (median not reached).

Half of the patients whose brain and spinal cord tumors responded to WELIREG had a response at 3.1 months or shorter. The range for time to response was from 2.5 to 11 months.

73% of these patients (11 out of 15) were still responding to treatment for 12 months or more.

Pancreas (pancreatic neuroendocrine tumors)

At the time of patient follow-up:

83% of patients (10 out of 12) saw tumors in their pancreas partially or completely shrink. 8 out of these 12 patients saw their tumors partially shrink and 2 saw their tumors completely shrink.

  • Patients who responded to treatment continued to respond from 11+ to 19+ months (median not reached).

Half of the patients whose pancreatic tumors responded to WELIREG had a response at 8.1 months or shorter. The range for time to response was from 2.7 to 11 months.

50% of these patients (5 out of 10) were still responding to treatment for 12 months or more.

Learn more about side effects for WELIREG

View side effects

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WELIREG is a prescription medicine used to treat von Hippel-Lindau (VHL) disease in adults who need treatment for a type of kidney cancer called renal cell carcinoma (RCC), tumors in the brain and spinal cord called central nervous system (CNS) hemangioblastomas, or a type of pancreatic cancer called pancreatic neuroendocrine tumors (pNET), that do not need surgery right away.

It is not known if WELIREG is safe and effective in children with VHL.

WELIREG can cause serious side effects. WELIREG can cause harm to your unborn baby.

  • A pregnancy test will be done before you start treatment.
  • Birth control methods that contain hormones may not work as well during treatment.
  • Females and males with female partners who can become pregnant should use an effective form of non-hormonal birth control (contraception) during treatment and for 1 week after your last dose.

WELIREG can cause serious side effects, including:

Low red blood cell counts (anemia). Low red blood cell counts can be severe. You may need a blood transfusion if your red blood cell counts drop too low. Your health care provider will do blood tests to check your red blood cell counts before you start and during treatment with WELIREG. Tell your health care provider if you get any symptoms of low red blood cell counts, including tiredness, feeling cold, shortness of breath, chest pain, or fast heartbeat.

Low oxygen levels in your body. WELIREG can cause low oxygen levels in your body that can be severe and may require you to stop treatment with WELIREG, receive oxygen therapy, or be hospitalized. Your health care provider will monitor your oxygen levels before you start and during treatment with WELIREG. Tell your health care provider or get medical help right away if you get symptoms of low oxygen in your body, including shortness of breath or increased heart rate.

Harm to your unborn baby. Treatment with WELIREG during pregnancy can cause harm to your unborn baby.

Females who are able to become pregnant:

  • Your health care provider will do a pregnancy test before you start treatment with WELIREG.
  • You should use an effective form of non-hormonal birth control (contraception) during treatment with WELIREG and for 1 week after your last dose.
  • Birth control methods that contain hormones (such as birth control pills, injections, or transdermal system patches) may not work as well during treatment with WELIREG.
  • Talk to your health care provider about birth control methods that may be right for you during treatment with WELIREG.
  • Tell your health care provider right away if you become pregnant or think you may be pregnant during treatment with WELIREG.

Males with female partners who are able to become pregnant:

  • You should use effective birth control (contraception) during treatment with WELIREG and for 1 week after your last dose.
  • Tell your health care provider right away if your partner becomes pregnant or thinks she is pregnant while you are taking WELIREG.

Before taking WELIREG, tell your health care provider about all of your medical conditions, including if you:

  • have low red blood cell counts (anemia)
  • are pregnant or plan to become pregnant
  • are breastfeeding or plan to breastfeed. It is not known if WELIREG passes into your breast milk. Do not breastfeed during treatment with WELIREG and for 1 week after your last dose.

Tell your health care provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking WELIREG with certain other medicines can affect each other and cause serious side effects and may affect the way certain other medicines work.

The most common side effects of WELIREG in adults with VHL disease include:

  • low red blood cell counts
  • tiredness
  • increased blood creatinine (kidney function test)
  • headache
  • dizziness
  • increased blood sugar (glucose) levels
  • nausea

The most common side effects of WELIREG when taken alone in adults with advanced ccRCC include:

  • low red blood cell counts
  • tiredness
  • muscle and joint pain
  • increased blood creatinine (kidney function test)
  • decreased white blood cell counts
  • increased blood liver enzymes (liver function tests)
  • decreased blood salts (sodium) levels
  • increased blood potassium levels

Your health care provider may change your dose, temporarily stop, or permanently stop treatment with WELIREG if you get certain side effects.

WELIREG may cause fertility problems in males and females, which may affect your ability to have children. Talk to your health care provider if this is a concern for you.

These are not all of the possible side effects of WELIREG. Call your health care provider for medical advice about side effects.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Please read the accompanying Medication Guide for WELIREG, including an important warning about harm to an unborn baby, and discuss it with your doctor. The physician Prescribing Information also is available.

Indication

WELIREG is a prescription medicine used to treat von Hippel-Lindau (VHL) disease in adults who need treatment for a type of kidney cancer called renal cell carcinoma (RCC), tumors in the brain and spinal cord called central nervous system (CNS) hemangioblastomas, or a type of pancreatic cancer called pancreatic neuroendocrine tumors (pNET), that do not need surgery right away.

It is not known if WELIREG is safe and effective in children with VHL.

Important Safety Information

WELIREG can cause serious side effects. WELIREG can cause harm to your unborn baby.

  • A pregnancy test will be done before you start treatment.
  • Birth control methods that contain hormones may not work as well during treatment.
  • Females and males with female partners who can become pregnant should use an effective form of non-hormonal birth control (contraception) during treatment and for 1 week after your last dose.

WELIREG can cause serious side effects, including:

Low red blood cell counts (anemia). Low red blood cell counts can be severe. You may need a blood transfusion if your red blood cell counts drop too low. Your health care provider will do blood tests to check your red blood cell counts before you start and during treatment with WELIREG. Tell your health care provider if you get any symptoms of low red blood cell counts, including tiredness, feeling cold, shortness of breath, chest pain, or fast heartbeat.

Low oxygen levels in your body. WELIREG can cause low oxygen levels in your body that can be severe and may require you to stop treatment with WELIREG, receive oxygen therapy, or be hospitalized. Your health care provider will monitor your oxygen levels before you start and during treatment with WELIREG. Tell your health care provider or get medical help right away if you get symptoms of low oxygen in your body, including shortness of breath or increased heart rate.

Harm to your unborn baby. Treatment with WELIREG during pregnancy can cause harm to your unborn baby.

Females who are able to become pregnant:

  • Your health care provider will do a pregnancy test before you start treatment with WELIREG.
  • You should use an effective form of non-hormonal birth control (contraception) during treatment with WELIREG and for 1 week after your last dose.
  • Birth control methods that contain hormones (such as birth control pills, injections, or transdermal system patches) may not work as well during treatment with WELIREG.
  • Talk to your health care provider about birth control methods that may be right for you during treatment with WELIREG.
  • Tell your health care provider right away if you become pregnant or think you may be pregnant during treatment with WELIREG.

Males with female partners who are able to become pregnant:

  • You should use effective birth control (contraception) during treatment with WELIREG and for 1 week after your last dose.
  • Tell your health care provider right away if your partner becomes pregnant or thinks she is pregnant while you are taking WELIREG.

Before taking WELIREG, tell your health care provider about all of your medical conditions, including if you:

  • have low red blood cell counts (anemia)
  • are pregnant or plan to become pregnant
  • are breastfeeding or plan to breastfeed. It is not known if WELIREG passes into your breast milk. Do not breastfeed during treatment with WELIREG and for 1 week after your last dose.

Tell your health care provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking WELIREG with certain other medicines can affect each other and cause serious side effects and may affect the way certain other medicines work.

The most common side effects of WELIREG in adults with VHL disease include:

  • low red blood cell counts
  • tiredness
  • increased blood creatinine (kidney function test)
  • headache
  • dizziness
  • increased blood sugar (glucose) levels
  • nausea

The most common side effects of WELIREG when taken alone in adults with advanced ccRCC include:

  • low red blood cell counts
  • tiredness
  • muscle and joint pain
  • increased blood creatinine (kidney function test)
  • decreased white blood cell counts
  • increased blood liver enzymes (liver function tests)
  • decreased blood salts (sodium) levels
  • increased blood potassium levels

Your health care provider may change your dose, temporarily stop, or permanently stop treatment with WELIREG if you get certain side effects.

WELIREG may cause fertility problems in males and females, which may affect your ability to have children. Talk to your health care provider if this is a concern for you.

These are not all of the possible side effects of WELIREG. Call your health care provider for medical advice about side effects.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Please read the accompanying Medication Guide for WELIREG, including an important warning about harm to an unborn baby, and discuss it with your doctor. The physician Prescribing Information also is available.