Showing posts with label New Drug Application (NDA). Show all posts
Showing posts with label New Drug Application (NDA). Show all posts

Tuesday, October 11, 2022

BioLineRx Announces Submission of New Drug Application (NDA) to FDA for Motixafortide in Stem Cell Mobilization

BioLineRx Ltd.  announced the  submission of  its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Motixafortide in stem cell mobilization (SCM) for autologous bone marrow transplantation for multiple myeloma patients.

The NDA submission is based on the overwhelmingly positive top-line results from BioLineRx's GENESIS Phase 3 trial of Motixafortide on top of G-CSF (versus placebo on top of G-CSF) in stem cell mobilization for autologous bone marrow transplantation in multiple myeloma patients. The study met all primary and secondary endpoints with a very high degree of statistical significance (p<0.0001). The combination was also found to be safe and well tolerated.

"The submission of our first NDA is a significant milestone for our Company and gives us potential line of sight towards launching a product that we successfully developed for an indication in substantial need of more effective treatment options," stated Philip Serlin, Chief Executive Officer of BioLineRx. "Notably, ~90% of multiple myeloma patients in the GENESIS study went directly to transplantation after mobilizing the optimal number of stem cells following only one administration of Motixafortide and in only one apheresis session, compared to less than 10% of those receiving G-CSF alone. This high success rate has a substantial clinical benefit, especially when considering that new induction treatments are more effective than ever before but cause subsequent difficulty in mobilizing the target number of stem cells for transplantation. The high success rate may also confer significant benefits to transplant institutions through the more efficient use of apheresis units, where there is often a lack of available machines."

"The totality of data that we have compiled for Motixafortide in stem cell mobilization – both clinical and pharmacoeconomic – suggest that Motixafortide, if approved, can quickly become the key component of a new standard of care on top of G-CSF for all multiple myeloma patients undergoing autologous stem cell transplantation. The submission of our NDA brings us one critical step closer to that goal, and we look forward to working closely with the FDA during its review process," Mr. Serlin concluded.

The FDA's decision on acceptance of BioLineRx's NDA filing is expected in November. Assuming the filing is accepted, the potential PDUFA date would be in Q2 2023 (under a priority review process, if applicable) or Q3 2023 (under a standard review process). As BioLineRx finalizes its commercialization plans for Motixafortide in the U.S., the Company continues to advance critical pre-launch activities, required under any commercialization scenario, to ensure a robust and targeted commercial launch very soon after its PDUFA date, assuming FDA approval.

Monday, December 27, 2021

Amylyx Pharmaceuticals Submits New Drug Application (NDA) for AMX0035 for the Treatment of ALS


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Amylyx Pharmaceuticals, Inc.  announced that, it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for AMX0035 (sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine)) for the treatment of amyotrophic lateral sclerosis (ALS).

“We are excited to share with the ALS community the exciting milestone that we have submitted our NDA to the FDA for review,” said Justin Klee, Co-CEO, Director and Co-Founder of Amylyx. Joshua Cohen, Co-CEO, Chairman and Co-Founder of Amylyx added, “Our team has worked and continues to work around the clock as we know time is of the essence for people living with ALS and their families. We will continue to keep the community closely updated on our progress.”
“We will continue to work closely with the FDA throughout the review process to move AMX0035 toward a potential regulatory approval as quickly and efficiently as possible,” said Tammy Sarnelli, Global Head of Regulatory Affairs of Amylyx. “We are so inspired by the people who participated in CENTAUR, the trial investigators, the ALS community and our partners and team, and we will continue to work tirelessly on behalf of you all.”

The NDA submission to the FDA is based on data from the CENTAUR trial, a placebo-controlled study evaluating 137 people with ALS. In this study, participants receiving AMX0035 had statistically significant slowing of functional decline at the end of the 6-month randomized phase as measured by the Revised ALS Functional Rating Scale (ALSFRS-R), the most commonly used scale in clinics worldwide to measure function in ALS. In an interim survival analysis conducted in all randomized participants from CENTAUR who were followed for up to three years, which included participants who continued to receive AMX0035 in an open-label extension phase during the follow-up period, participants who started on AMX0035 during the placebo-controlled phase of CENTAUR showed a 44% lower risk of death compared to those who started on placebo during the placebo-controlled phase (HR 0.56; 95% CI, 0.34-0.92). Median survival duration through the open-label long-term follow-up phase was 25.0 months (95% CI, 19.0-33.6 months) in the group that started on AMX0035 and 18.5 months (95% CI, 13.5-23.2 months) in the group that started on placebo, a 6.5-month difference. Overall, reported rates of adverse events and discontinuations were substantially similar between AMX0035 and placebo groups during the 24-week randomized phase; however, gastrointestinal events occurred with greater frequency (≥2%) in the AMX0035 group. Detailed data from CENTAUR is published in the New England Journal of Medicine (NEJM) and Muscle and Nerve.

“This submission brings hope to people living with ALS and their families and caregivers,” said Merit Cudkowicz, M.D., co-principal investigator of the CENTAUR trial and co-founder of the Northeast ALS Consortium, Director of the Healey & AMG Center for ALS and Chair of Neurology at Massachusetts General Hospital and the Julieanne Dorn Professor of Neurology at Harvard Medical School. “We are honored to have led the collaboration between the Healey & AMG Center for ALS, ALS Finding a Cure, the ALS Association, and NEALS that made the CENTAUR trial a reality, the efforts and results of which made this NDA submission possible.”

“For people living with ALS and their physicians, this is a significant development offering hope of a potential new treatment option that has been shown to slow ALS disease progression and extend the time families that face this life-threatening disease have together,” said Sabrina Paganoni, M.D., Ph.D., principal investigator of the CENTAUR trial, investigator at the Healey & AMG Center for ALS at Massachusetts General Hospital and Assistant Professor of PM&R at Harvard Medical School and Spaulding Rehabilitation Hospital.

As previously reported, Amylyx filed a New Drug Submission (NDS) for AMX0035 for the treatment of ALS with Health Canada in June 2021. Amylyx also intends to submit a Marketing Authorization Application (MAA) for AMX0035 to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) by approximately the end of 2021 and to initiate a global Phase 3 clinical trial with sites in Europe and the United States in the fourth quarter of 2021. The Phase 3 PHOENIX trial of AMX0035 for the treatment of people with ALS will assess the safety and efficacy of AMX0035 in an international population of approximately 600 participants and build upon findings from the CENTAUR trial. Amylyx is currently exploring the possibility of an Expanded Access Program (EAP) in the United States. If implemented, the EAP would run in parallel with the ongoing Phase 3 PHOENIX trial and marketing application reviews. Further information about the EAP is expected in the fourth quarter of 2021.


Monday, May 1, 2017

Eisai Inc. and Arena Pharmaceuticals Announce FDA Approval of Belviq XR (lorcaserin HCl) Extended-Release Tablets

In continuation of my update on Lorcaserin
Eisai Inc. and Arena Pharmaceuticals, Inc.   announced   the approval by  U.S. Food and Drug Administration (FDA)  the New Drug Application (NDA) for Belviq XR (lorcaserin HCl) CIV extended-release 20 mg tablets. The new formulation of lorcaserin will offer patients a once-a-day dosing option that may help them achieve and maintain weight loss. Belviq XR is expected to be available in the fall of 2016. In connection with the approval, Arena will receive a $10 million milestone payment.
Belviq XR is proven to be slowly absorbed in the body and lasts throughout the day. Both the original 10 mg twice-daily formulation of Belviq and newly-approved 20 mg once-daily extended release formulation are approved for use with a reduced-calorie diet and increased physical activity for chronic weight management in adults who have a body mass index (BMI) of 30 kg/m2 or greater (obese), or BMI of 27 kg/m2 or greater (overweight) with at least one weight-related medical condition, such as high blood pressure, high cholesterol, or type 2 diabetes. It is not known if Belviq or Belviq XR, when taken with other prescription, over-the-counter, or herbal weight-loss products, is safe and effective. It is not known if Belviq or Belviq XR changes your risk of heart problems, stroke, or death due to heart problems or stroke.
"With approximately two-thirds of the U.S. population living with extra weight or obesity, there is a significant and growing need to address chronic weight management," said Louis J. Aronne, M.D., Director of the Comprehensive Weight Control Center at Weill Cornell Medicine, physician at NewYork-Presbyterian/Weill Cornell Medical Center and Principal Investigator of the BELVIQ clinical trials. "Having a once-daily treatment may offer an option for patients to stay on track to meet their weight loss goals."
The bioequivalence and bioavailability of once-daily Belviq XR 20 mg compared with twice-daily Belviq 10 mg was based on two Phase 1 registrational clinical trials among healthy adult subjects. The most common treatment-emergent adverse events were similar to those seen in the Phase 3 clinical trials of Belviq 10 mg twice-daily.
"We're excited to offer this once-a-day option of lorcaserin," said Andrew Satlin, M.D., Executive Vice President, Neurology Business Group, Eisai Inc. "This option may provide another choice for patients who are overweight or obese and find it difficult to lose weight through diet and exercise alone. The development of this new formulation further underscores Eisai's ongoing commitment to help address the health care needs of this underserved population."
"We are pleased that once-daily Belviq XR has been approved by the FDA and will provide patients another option for weight loss," said Amit D. Munshi, Arena's President and Chief Executive Officer. "The approval of this new formulation is another example of Arena's success in supporting our collaborators."

What are Belviq and Belviq XR?

Belviq and Belviq XR are FDA-approved prescription weight-loss medications that, when used with diet and exercise, can help some overweight (Body Mass Index [BMI] ≥27 kg/m2) adults with a weight-related medical problem, or obese (BMI ≥30 kg/m²) adults, lose weight and keep it off.
It is not known if Belviq or Belviq XR when taken with other prescription, over-the-counter, or herbal weight-loss products is safe and effective. It is not known if Belviq or Belviq XR changes your risk of heart problems, stroke, or death due to heart problems or stroke.

Tuesday, January 10, 2017

FDA Issues Complete Response Letter for Apadaz New Drug Application

KemPharm, Inc.  a clinical-stage specialty pharmaceutical company engaged in the discovery and development of proprietary prodrugs,  announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) regarding the New Drug Application (NDA) for Apadaz™ (benzhydrocodone and acetaminophen), KemPharm’s investigational abuse-deterrent product candidate for the short-term management of acute pain.
Benzhydrocodone.svg benzhydrocodone        Paracetamol-skeletal.svg acetaminophen

The FDA issues CRLs to indicate that the Agency considers the review cycle for an application is complete and that the application is not ready for approval in its present form. Included in the CRL is guidance that describes all specific deficiencies that the FDA has identified in the application. When possible, the FDA recommends actions that the applicant may take to place the application in condition for approval.
“After last week’s amendment request, a Complete Response Letter from the FDA was received for the Apadaz NDA,” said Travis C. Mickle, Ph.D., President and CEO of KemPharm. “We are currently evaluating the points raised in the CRL and intend to request an End of Review meeting with the Agency to determine the pathway forward for Apadaz.”

Thursday, May 2, 2013

FDA Approves Tris Pharma's New Drug Application for Karbinal ER

In continuation of my update on Karbinal (carbinoxamine maleate)...

FDA, has approved its New Drug Application (NDA) for Karbinal ER (carbinoxamine maleate) Extended-release Oral Suspension 4mg/5mL, the first sustained-release histamine receptor blocking agent indicated for the treatment of seasonal and perennial allergic rhinitis in children ages 2 and up.

Monday, December 17, 2012

Pfizer, Ligand announce FDA acceptance of bazedoxifene/conjugated estrogens NDA

Pfizer Inc.  and Ligand Pharmaceuticals Incorporated recently,  announced that the United States Food and Drug Administration (FDA) accepted for review a New Drug Application (NDA) for bazedoxifene/conjugated estrogens (BZA/CE), a potential new medicine for non-hysterectomized women for the treatment of moderate-to-severe vasomotor symptoms (VMS) and vulvar and vaginal atrophy (VVA) associated with menopause, as well as the prevention of postmenopausal osteoporosis. The FDA Prescription Drug User Fee Act (PDUFA) date is October 3, 2013. 

Pfizer, Ligand announce FDA acceptance of bazedoxifene/conjugated estrogens NDA