UPSHER-SMITH PRESENTS EXPANDED RARE DISEASE PORTFOLIO AT 2024 CHILD NEUROLOGY SOCIETY ANNUAL MEETING

Visit Our Booths to Learn More about the Company’s Latest Product Offerings and Promise of Support™ Program

MAPLE GROVE, Minn., Nov. 11, 2024 /PRNewswire/ -- Upsher-Smith Laboratories, LLC (Upsher-Smith) today announced that it will attend the 2024 Child Neurology Society (CNS) Annual Meeting in San Diego, CA from November 11-14, 2024. Representatives from Upsher-Smith will be at our booths to speak with clinicians about its expanding portfolio of medications including newly acquired VIGAFYDE™ (vigabatrin) Oral Solution and recently launched TORPENZ™ (everolimus) Tablets, which became available in August 2024.

VIGAFYDE^™ is the first and only ready-to-use vigabatrin oral solution and represents a welcome expansion of options for the treatment of infantile spasms as the first new drug application approved for this condition in 15 years. As the only premixed, ready-to-use vigabatrin formulation, VIGAFYDE™ offers patients and caregivers a new option in liquid vigabatrin that does not require reconstitution by the caregiver. VIGAFYDE^™, which became available to patients in September 2024, is indicated as monotherapy for the treatment of infantile spasms in pediatric patients 1 month to 2 years, where the potential benefits outweigh the potential risk of vision loss.

TORPENZ™, an oral mTOR inhibitor used to treat certain manifestations of Tuberous Sclerosis Complex (TSC), is the only everolimus backed by the Upsher-Smith Promise of Support™ Program to deliver reliable and consistent support to patients, caregivers, and healthcare providers in the TSC community. TORPENZ™ is indicated for the treatment of adult patients with renal angiomyolipoma and TSC, not requiring immediate surgery and is also indicated in adult and pediatric patients aged 1 year and older with TSC for the treatment of subependymal giant cell astrocytoma (SEGA) that requires therapeutic intervention but cannot be curatively resected.

“Since the successful launch of VIGADRONE^® (vigabatrin) for Oral Solution to the specialty pharmacy channel in 2018, we have broadened our offerings to include VIGADRONE^® (vigabatrin) Tablets, TORPENZ™ Tablets, and now VIGAFYDE™ ready-to-use solution,” said Jim Maahs, Head of Commercial, Upsher-Smith. “As part of our commitment to supporting the rare disease community, Upsher-Smith is dedicated to not only maintaining, but enhancing the support and resources available to patients, caregivers, and healthcare practitioners. We encourage attendees to stop by our booths during this year’s CNS meeting to learn more.”

About Upsher-Smith’s Promise of Support™ Program

Central to Upsher-Smith’s commitment to rare disease communities is its Promise of Support™ Program, ensuring comprehensive assistance for patients, caregivers, and healthcare providers throughout the treatment journey. For nearly a decade, the program has offered resources for uninterrupted medication supply, prior authorization support, patient education materials, collaboration with a leading specialty pharmacy partner, and streamlined prescribing and enrollment procedures. It also includes copay assistance for eligible patients to help lessen the financial burden on patients and their families.

To learn more about Upsher-Smith, its products, and the Promise of Support™ Program visit: www.upsher-smith.com.

Vigabatrin REMS

All vigabatrin products are governed by a Risk Evaluation and Mitigation Strategy (REMS) mandated by the FDA. The Vigabatrin REMS ensures that patients and healthcare providers make informed risk-benefit decisions about its use.

Healthcare providers must be enrolled in the Vigabatrin REMS to prescribe these products, and patients must be enrolled to receive them.

IMPORTANT SAFETY INFORMATION FOR VIGAFYDE™ (vigabatrin)

What is the most important information I should know about VIGAFYDE?

VIGAFYDE can cause serious side effects, including:

• Permanent vision loss: VIGAFYDE can damage the vision of anyone who takes it. Some babies can have severe loss, particularly to their ability to see to the side when they look straight ahead (peripheral vision). With severe vision loss, your baby may only be able to see things straight in front of them (sometimes called “tunnel vision”). Your baby also may have blurry vision. If this happens, it will not get better.

Tell your healthcare provider right away if your baby might not be seeing as well as before starting VIGAFYDE or is acting differently than normal.

Vision loss in babies: Because of the risk of vision loss, VIGAFYDE is used in babies 1 month to 2 years of age with infantile spasms only when you and your healthcare provider decide that the possible benefits of VIGAFYDE are more important than the risks.

• Parents or caregivers are not likely to recognize the symptoms of vision loss in babies until it is severe. Healthcare providers may not find vision loss in babies until it is severe.

• It is difficult to test vision in babies, but, to the extent possible, all babies should have their vision tested before starting VIGAFYDE or within 4 weeks after starting VIGAFYDE, and every 3 months after that until VIGAFYDE is stopped. Your baby also should have a vision test about 3 to 6 months after VIGAFYDE is stopped.

• Your healthcare provider will determine if your baby can be tested. If your baby cannot be tested, your healthcare provider may continue prescribing VIGAFYDE, but they will not be able to watch for any vision loss.

Even if your baby’s vision seems fine, it is important to get regular vision tests because damage can happen before your baby acts differently. Even these regular vision exams may not show the damage to your baby’s vision before it is severe and permanent.

All babies who take VIGAFYDE:

• Are at risk for permanent vision loss with any amount of VIGAFYDE.

• The risk of vision loss may increase with higher doses of VIGAFYDE, and the longer it is taken.

• It is not possible for your healthcare provider to know when vision loss will happen. It could happen soon after starting VIGAFYDE or any time during treatment. It may even happen after treatment has stopped.

Because VIGAFYDE might cause permanent vision loss, it is available to healthcare providers and patients only under a special program called Vigabatrin Risk Evaluation and Mitigation Strategy (REMS). Your healthcare provider will explain the details of the Vigabatrin REMS to you. For more information, go to or call 1-866-244-8175.

• Magnetic resonance imaging (MRI) changes in babies with infantile spasms (IS): brain pictures taken by MRI show changes in some babies after they are given VIGAFYDE. It is not known if these changes are harmful.

What are the possible side effects of VIGAFYDE?

VIGAFYDE can cause serious side effects, including sleepiness (sleepy babies may have a harder time suckling and feeding or may be irritable) and weight gain without swelling. Serious side effects that happen in adults who take vigabatrin and are not known if they will happen in babies are low red blood cell counts, nerve problems, and swelling.

Tell your baby’s healthcare provider right away if your baby’s seizures get worse.

The most common side effects of VIGAFYDE in babies include sleepiness, ear infection, swelling of the bronchial tubes, and irritability.

Tell your healthcare provider if your baby has any side effect that bothers them or that does not go away. These are not all the possible side effects of VIGAFYDE.

Do not stop taking VIGAFYDE suddenly. This can cause serious problems. Talk to your healthcare provider for directions on how to stop treatment.

What should I tell my healthcare provider before starting VIGAFYDE?

Before giving VIGAFYDE to your baby for IS, inform your healthcare provider about all your baby’s medical conditions. This includes whether your baby has ever had an allergic reaction to vigabatrin, such as hives, itching, or trouble breathing, as well as any history of vision problems, or kidney issues. Tell your healthcare provider about all the medicines your baby takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Interactions between VIGAFYDE and other medicines could lead to side effects.

You may report side effects to the FDA at http://www.fda.gov/medwatch or call 1-800-FDA-1088 or Pyros at 1-855-406-1010.

Please see full Prescribing Information, including BOXED WARNING, Medication Guide, and Instructions for Use for additional Important Safety Information.

WHAT IMPORTANT SAFETY INFORMATION SHOULD I KNOW ABOUT TORPENZ™ (everolimus) TABLETS in Tuberous Sclerosis Complex?

Do not take TORPENZ if you have had an allergic reaction to everolimus. Talk to your healthcare provider before taking TORPENZ if you are allergic to sirolimus or temsirolimus. Ask your provider if you do not know.

TORPENZ can cause serious side effects, including:

• Lung or breathing problems. In some people, these may be severe and can be life threatening. Tell your healthcare provider right away if you have new or worsening cough, shortness of breath, chest pain, difficulty breathing or wheezing.

• Higher likelihood of infection such as pneumonia, or a bacterial, fungal, or viral infection. Viral infections may include active hepatitis B in people who have had hepatitis B in the past (reactivation). In some adults and children, these infections may be severe and can be life threatening. You may need to be treated as soon as possible. Tell your healthcare provider right away if you have a temperature of 100.5˚F or above, chills, or do not feel well. Symptoms of infection may include:

• Severe allergic reactions. Get medical help right away if you have signs of an allergic reaction, including rash, itching, hives, flushing, trouble breathing or swallowing, chest pain or dizziness.

• Possible increased risk for a type of allergic reaction called angioedema in people who take an Angiotensin-Converting Enzyme (ACE) inhibitor with TORPENZ. Talk with your healthcare provider before taking TORPENZ if you are not sure if you take an ACE inhibitor. Get medical help right away if you have trouble breathing or develop swelling of your tongue, mouth, or throat during treatment with TORPENZ.

• Mouth ulcers and sores. Mouth ulcers and sores are common during treatment with TORPENZ but can also be severe. When you start TORPENZ, your healthcare provider may tell you to also start a prescription mouthwash to reduce the likelihood of getting mouth ulcers or sores and to reduce their severity. Follow your provider’s instructions on how to use this mouthwash. If you develop pain, discomfort, or open sores in your mouth, tell your provider.

• Kidney failure. In some people, this may be severe and can be life threatening. Your healthcare provider should check your kidney function before and during treatment.

• Wound healing problems. Wounds may not heal properly during TORPENZ treatment. Tell your healthcare provider if you plan to have any surgery before starting or during treatment. You should stop TORPENZ at least 1 week before planned surgery. Your provider should tell you when to start taking TORPENZ again after surgery.

• Increased blood sugar and fat (cholesterol and triglyceride) levels in the blood. Your healthcare provider should check your fasting blood sugar, cholesterol, and triglyceride levels in the blood before you start and during treatment.

• Decreased blood cell counts. TORPENZ can cause you to have decreased red blood cells, white blood cells and platelets. Your healthcare provider should check your blood cell counts before you start and during treatment.

• Worsening side effects from radiation treatment that can sometimes be severe. Tell your healthcare provider if you have had or are planning to receive radiation therapy.

Before taking TORPENZ, tell your healthcare provider about all of your medical conditions, including if you:

• Have or have had kidney or liver problems, have diabetes or high blood sugar, have high blood cholesterol, have any infections, or previously had hepatitis B.

• Are scheduled to receive any vaccinations. You should not receive a “live vaccine” or be around people who have recently received a “live vaccine” during your treatment with TORPENZ. If you are not sure about the type of immunization or vaccine, ask your provider. For children, work with your provider to complete the recommended childhood series of vaccines before your child starts TORPENZ.

• Are pregnant, can become pregnant, or have a partner who can become pregnant. TORPENZ can cause harm to your unborn baby. For females who are able to become pregnant, your provider will give you a pregnancy test before you start TORPENZ. Use effective birth control during treatment and for 8 weeks after your last dose of TORPENZ. Males with a female partner should use effective birth control during treatment and for 4 weeks after your last dose of TORPENZ.

• Are breastfeeding or plan to breastfeed. Do not breastfeed during treatment and for 2 weeks after your last dose of TORPENZ.

• Are planning to have or have had a recent surgery. You should stop taking TORPENZ at least 1 week before planned surgery.

• Have received or are planning to receive radiation therapy.

Tell your healthcare provider about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking TORPENZ with some medicines can cause serious side effects. Keep a list of medications you take, and show it to your healthcare provider and pharmacist when you get a new medicine. Especially tell your provider if you take:

• St. John’s Wort (Hypericum perforatum)

• Medicine for fungal or bacterial infections, tuberculosis, seizures, HIV-AIDS, heart conditions or high blood pressure, including ACE inhibitors

• Medicines that weaken your immune system (your body’s ability to fight infections and other problems)

Ask your provider or pharmacist if you are not sure if your medicine is one of those taken for the conditions listed above. If you are taking any medicines for the conditions listed above, your provider might need to prescribe a different medicine or your dose of TORPENZ may need to be changed. Tell your provider before you start any new medicine.

You should not drink grapefruit juice or eat grapefruit during your treatment with TORPENZ. It may make the amount of TORPENZ in your blood increase to a harmful level.

The most common side effect of TORPENZ in people who have SEGA or renal angiomyolipoma include respiratory tract infection.

Other side effects that may occur with TORPENZ:

• Absence of menstrual periods (menstruation). Tell your healthcare provider if this happens.

• TORPENZ may affect fertility and may affect your ability to become pregnant if you are female or your ability to father a child if you are male. Talk to your provider if this is a concern.

Tell your healthcare provider if you have any side effect that bothers you or does not go away.

These are not all the possible side effects of TORPENZ. For more information, ask your healthcare provider or pharmacist. Call your doctor for medical advice about side effects. You can also visit TORPENZ.com, upsher-smith.com or call 1-888-650-3789.

You are encouraged to report negative side effects of prescription drugs to FDA. Visit www.fda.gov/medwatch or call 1-800-332-1088.

WHAT ARE TORPENZ (everolimus) TABLETS?

TORPENZ is a prescription medicine used to treat the following types of benign (non-cancerous) tumors that are seen with a genetic condition called tuberous sclerosis complex (TSC):

• Brain tumors called subependymal giant cell astrocytoma (SEGA), when the tumor cannot be removed completely by surgery, in adults and children 1 year and older.
• Kidney tumors called angiomyolipoma, when the tumor does not require surgery right away, in adults.

For additional information, including safety information, for non-TSC indications, see the full Prescribing Information, including Patient Information.

WHAT IMPORTANT SAFETY INFORMATION SHOULD I KNOW ABOUT VIGADRONE^® (vigabatrin)?

• Because

  VIGADRONE might cause permanent vision loss, it is available to healthcare providers and patients only under a special program called the Vigabatrin Risk Evaluation and Mitigation Strategy (REMS) Program. Your healthcare provider will explain the details of this Program to you.

• VIGADRONE can damage the vision of anyone who takes it. Some people can have severe loss, particularly to their ability to see to the side when looking straight ahead (peripheral vision). With severe vision loss, you may only be able to see things straight in front of you (sometimes called “tunnel vision”). You may also have blurry vision. If this happens, it will not get better.

• Tell your healthcare provider right away if you (or your child): might not be seeing as well as before starting VIGADRONE; start to trip, bump into things, or are more clumsy than usual; are surprised by people or things coming in front of you that seem to come out of nowhere; or if your baby is acting differently than normal.

  These changes can mean that vision damage has occurred.

• Regular vision testing is recommended. It is recommended that your healthcare provider test your (or your child’s) vision before or within 4 weeks after starting VIGADRONE, and at least every 3 months during treatment until VIGADRONE is stopped. It is also recommended that vision be tested about 3 to 6 months after VIGADRONE is stopped. It is difficult to test vision in babies, but to the extent possible, all patients should have their vision tested. Your healthcare provider will determine if testing can be done.

  Regular vision testing is important because damage can happen before any changes are noticed.

• Vision tests cannot prevent the vision damage that can happen with VIGADRONE, but they do allow VIGADRONE to be stopped if vision has gotten worse, which usually will lessen further damage. Even these regular vision tests may not show vision damage before it is serious and permanent.

  Parents, caregivers, and healthcare providers may not recognize the symptoms, or find vision loss in patients, until it is severe.

• If you do not have these vision tests regularly, your healthcare provider may stop prescribing

  VIGADRONE for you (or your child). Some people are not able to complete vision testing. If vision testing cannot be done, your healthcare provider may continue prescribing VIGADRONE, but will not be able to watch for any vision loss.

• Magnetic resonance imaging (MRI) changes in patients with infantile spasms (IS). Brain pictures taken by MRI show changes in some patients after they are given VIGADRONE. It is not known if these changes are harmful.

• A type of swelling in the brain called intramyelinic edema (IME) has been seen in autopsy examination of patients treated with vigabatrin.

• Risk of suicidal thoughts or actions. Like other antiepileptic drugs, VIGADRONE may cause suicidal thoughts and actions in some people (about 1 in 500 people). Call a healthcare provider right away if you (or your child) have any symptoms, especially sudden changes in mood, behaviors, thoughts or feelings, and especially if they are new, worse, or worry you.

• Do not stop VIGADRONE without first talking to a healthcare provider. Stopping VIGADRONE suddenly can cause seizures that will not stop.

VIGADRONE can cause serious side effects such as low red blood cell counts (anemia), sleepiness and tiredness, nerve problems, weight gain, and swelling. Because VIGADRONE causes sleepiness and tiredness, do not drive, operate machinery, or perform any hazardous task, unless it is decided that these things can be done safely. VIGADRONE may make certain types of seizures worse. Tell your healthcare provider right away if seizures get worse.

Before starting VIGADRONE, tell your doctor about all of your (or your child’s) medical conditions including depression, mood problems, suicidal thoughts or behavior, any allergic reaction to VIGADRONE, vision problems, kidney problems, low red blood cell counts (anemia), and any nervous or mental illnesses. Tell your doctor about all the medicines you (or your child) take.

If you are breastfeeding or plan to breastfeed, VIGADRONE can pass into breast milk and may harm your baby. Breastfeeding is not recommended.

If you are pregnant or plan to become pregnant, VIGADRONE can cause harm to your unborn baby. You and your healthcare provider will have to decide if you should take VIGADRONE while you are pregnant.

The most common side effects of VIGADRONE in adults include: blurred vision, sleepiness, dizziness, problems walking or feeling uncoordinated, shaking (tremor) and tiredness.

The most common side effect of VIGADRONE in children 3 to 16 years of age is weight gain. Also expect side effects like those seen in adults.

The most common side effects of VIGADRONE in babies include: sleepiness (sleepy babies may have a harder time suckling and feeding or may be irritable), swelling in the bronchial tubes (bronchitis), ear infection and irritability.

Tell your healthcare provider if you or your child have any side effect that bothers you or that does not go away.

This is the most important information to know about VIGADRONE, but it is not all the safety information. For more information, ask your healthcare provider or pharmacist, or please see the VIGADRONE Medication Guide, full Prescribing Information including Boxed Warning for risk of permanent vision loss, and Instructions for Use. You can also visit VIGADRONE.com, upsher-smith.com or call 1-888-650-3789.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-332-1088.

WHAT IS VIGADRONE? VIGADRONE^® (vigabatrin) is a prescription medicine used to treat:

• Infantile Spasms (IS) in patients 1 month to 2 years of age, if you and your healthcare provider decide the possible benefits of taking VIGADRONE are more important than the possible risk of vision loss.
• Refractory Complex Partial Seizures (CPS) in adults and children 2 years and older with refractory complex partial seizures (CPS) along with other treatments if:
• The CPS do not respond well enough to several other treatments, and
• You and your healthcare provider decide the possible benefit of taking VIGADRONE is more important than the risk of vision loss.

VIGADRONE should not be the first medicine used to treat CPS.

About Upsher-Smith

Upsher-Smith Laboratories, LLC, now a member of Bora Group, is a trusted U.S. pharmaceutical company that strives to improve the health and lives of patients through an unwavering commitment to high-quality products and sustainable growth. We bring generics and brands to a wide array of customers, always backed by our attentive level of service, strong industry relationships, and dedication to uninterrupted supply. For more information, visit www.upsher-smith.com.

About Bora

Founded in 2007, Bora Pharmaceutical Co., Ltd. (“Bora” or “the Company”, 6472.TW) now is the largest pharmaceutical manufacturer in Taiwan with well-connected global distribution to supply more than 100 countries around the world. Bora is dedicated to becoming a global leader in pharmaceutical manufacturing by offering its clients the best quality, efficiency and reliability. For more information, visit www.bora-corp.com.

TORPENZ, VIGADRONE, and Promise of Support are trademarks of Upsher-Smith Laboratories, LLC.

VIGAFYDE is a trademark of Pyros Pharmaceuticals, Inc.

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