Ipsen’s Onivyde Regimen, a Potential New Standard-of-Care First-Line Therapy in Metastatic Pancreatic Adenocarcinoma, Approved by FDA

Ipsen (Euronext: IPN; ADR: IPSEY) announced today that the U.S. Food and Drug Administration (FDA) has approved the supplemental new drug application for Onivyde (irinotecan liposome injection) plus oxaliplatin, fluorouracil and leucovorin (NALIRIFOX) as a first-line treatment in adults living with metastatic pancreatic adenocarcinoma (mPDAC). This is the second approval for an Onivyde regimen in mPDAC, following the FDA’s approval in 2015 of Onivyde plus fluorouracil and leucovorin following disease progression with gemcitabine-based therapy.

“The results from the Phase III NAPOLI 3 trial represent the first positive data for an investigational regimen in first-line metastatic pancreatic adenocarcinoma versus the currently approved nab-paclitaxel and gemcitabine regimen,” said Christelle Huguet, EVP and Head of Research and Development, Ipsen. “With today’s approval, this Onivyde (NALIRIFOX) regimen can now offer a potential new standard-of-care treatment option with proven survival benefits for people living with metastatic pancreatic adenocarcinoma in the U.S.”

Pancreatic adenocarcinoma (PDAC) is the most common type of cancer that forms in the pancreas, with more than 60,000 people diagnosed in the U.S. each year and nearly 500,000 people globally.3,4 Since there are no specific symptoms in the early stages, PDAC is often detected late and after the disease has spread to other parts of the body (metastatic or stage IV).5 Characterized as a complex cancer due to rapid tumor progression, limited genetic targets and multiple resistance mechanisms,6 mPDAC has a poor prognosis with fewer than 20% of people surviving longer than one year.4,5 Overall, pancreatic cancer has the lowest five-year survival rate of all cancer types globally and in the U.S.4,5

“Metastatic pancreatic adenocarcinoma is a difficult disease to manage with very few available treatment options. Given the reality of this aggressive form of cancer and the complexity of the disease, every advance in the treatment landscape represents a meaningful improvement in patient outcomes.” said Dr. Zev Wainberg, Professor of Medicine and Co-Director of the UCLA GI Oncology Program. “The approval of this Onivyde regimen is an important milestone for people living with mPDAC, their families and healthcare providers, with the NAPOLI 3 trial having demonstrated survival benefits versus a current standard of care treatment option.”

“We are pleased that the U.S. Food and Drug Administration has issued this new approval of the NALIRIFOX regimen. With each new approved treatment, there is more hope for those who will be diagnosed in the future and people currently living with pancreatic cancer may have more time with their loved ones,” said Julie Fleshman, JD, MBA, President and CEO of Pancreatic Cancer Action Network (PanCAN), a patient advocacy organization committed to providing evidence-based information and resources to patients and caregivers, along with advancing research to improve patient outcomes. “We are thankful to the patients who participated in this clinical trial as they play a crucial role in advancing treatments for pancreatic cancer.”

https://en.wikipedia.org/wiki/Irinotecan

Ipsen’s Onivyde Regimen, a Potential New Standard-of-Care First-Line Therapy in Metastatic Pancreatic Adenocarcinoma, Approved by FDA

Balversa (erdafitinib) Approved for Advanced Bladder Cancer

Balversa (erdafitinib) has been approved by the U.S. Food and Drug Administration to treat adults with advanced or spreading bladder cancer caused by a genetic defect called FGFR3 or FGFR2, the agency said in a news release.

"We're in an era of more personalized or precision medicine, and the ability to target cancer treatment to a patient's specific genetic mutation or biomarker is becoming the standard, with advances being made in new disease types. Today's approval represents the first personalized treatment targeting susceptible FGFR genetic alternations for patients with metastatic bladder cancer," said Dr. Richard Pazdur, director of the FDA's Oncology Center of Excellence.

Bladder cancer is the sixth most common cancer in the United States, the FDA said. About 20 percent of people with the disease have an FGFR mutation.

Balversa was clinically studied in a trial involving 87 people with advanced or metastatic bladder cancer and the FGFR3 or FGFR2 genetic mutation who hadn't responded to chemotherapy, the FDA said. About one-third of those given the drug had a complete or partial response to the medication; the average response lasting about 5 1/2 months.

Common side effects of Balversa included a jump in phosphate levels, mouth sores, feeling tired, changes in kidney function, diarrhea, dry mouth and changes in liver function.

Balversa may trigger serious eye problems, including inflamed eyes, inflamed cornea and disorders of the retina, the agency warned.

Doctors and other health professionals should tell men with female partners of child-bearing potential to use contraception during treatment with Balversa and for one month after the last dose, the FDA said. Women who are pregnant or breastfeeding shouldn't take Balversa because it may harm a developing fetus or newborn.

https://en.wikipedia.org/wiki/Erdafitinib

https://pubchem.ncbi.nlm.nih.gov/compound/Erdafitinib

Balversa (erdafitinib) Approved for Advanced Bladder Cancer 

Mayzent Approved for Relapsing MS

Mayzent (siponimod) pills have been approved by the U.S. Food and Drug Administration for adults with relapsing multiple sclerosis (MS).

"Multiple sclerosis can have a profound impact on a person's life," said Dr. Billy Dunn, director of the agency's Division of Neurology Products.

MS is an autoimmune disease of the central nervous system that disrupts communication between the brain and other parts of the body. Most people have initial symptoms between the ages of 20 and 40, and MS strikes more women than men, the FDA said in a news release.

Many people with MS are left permanently disabled and worsen over time, the agency said.

Mayzent's effectiveness was shown in clinical studies of 1,651 people. Progression of disability was significantly lower among those who took Mayzent than in a group that took a placebo. Mayzent also decreased the number of relapses, the FDA said.

The drug must be accompanied by a patient medication guide that describes the medication's uses and risks. Since Mayzent may raise the risk of infections, users should have a complete blood count before treatment starts. And users should contact their doctor if they have any vision changes, changes in heart rate or trouble breathing, the FDA said.

Since Mayzent may harm a developing fetus, women of childbearing age should use contraception during treatment and for 10 days after stopping the drug.

Mayzent's most common side effects include headache, high blood pressure and liver problems.

https://en.wikipedia.org/wiki/Siponimod

Mayzent Approved for Relapsing MS 

Mavenclad Approved for Multiple Sclerosis

In continuation of my update on Mavenclad (cladribine)

  

Mavenclad (cladribine) tablets have been approved by the U.S. Food and Drug Administration to treat relapsing multiple sclerosis (MS) in adults.

An agency news release said the drug is recommended for people who have had an inadequate response to, or are unable to tolerate, another medication approved for MS. Mavenclad is not sanctioned for a form of MS called clinically isolated syndrome.

MS is a chronic autoimmune disease of the central nervous system that disrupts communication between the brain and other parts of the body. Most people have initial symptoms between ages 20 and 40, more often in women than men. The disease progresses over time and typically leads to lifelong disability.

Mavenclad's effectiveness was shown in clinical studies involving 1,326 people with relapsing forms of MS who had least one relapse in the prior 12 months. The drug significantly decreased the number of relapses compared to a placebo, the FDA said.

Mavenclad must be dispensed with a patient medication guide that describes the drug's uses and risks. The medication's label includes a boxed warning of increased risk of cancer, worsening existing cancer, and fetal harm. The drug should not be used in pregnant women and among people of reproductive age who do not use contraception during treatment and for six months afterward, the FDA said. Mavenclad should be stopped if the user becomes pregnant.

Other significant warnings include a risk of a drop in white blood cells called lymphocytes, infections, bone marrow suppression and liver injury. The most common adverse reactions include upper respiratory tract infection and headache.

https://en.wikipedia.org/wiki/Cladribine

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Mavenclad Approved for Multiple Sclerosis