globenewswire | September 12, 2023
Aquestive Therapeutics, Inc. a pharmaceutical company advancing medicines to solve patients’ problems with current standards of care and provide transformative products to improve their lives, today announced acceptance by the U.S. Food and Drug Administration (FDA) of the Company’s NDA for Libervant™ (diazepam) Buccal Film in pediatric patients between two and five years of age and the assignment of a PDUFA goal date of April 28, 2024.
Aquestive’s NDA for approval of Libervant™ Buccal Film for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e., seizure clusters, acute repetitive seizures) in patients between two and five years of age has been accepted by the FDA. Diastat® (diazepam) Rectal Gel is the only treatment currently available to this patient population for this indication. The Company received tentative approval for Libervant for the treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e., seizure clusters, acute repetitive seizures) in patients 12 years of age and older in August 2022, but Libervant is currently under an orphan drug block to market access until January 2027.
“The FDA’s acceptance of our most recent filing for Libervant is another step forward in bringing this important treatment option to patients,” said Daniel Barber, Chief Executive Officer of Aquestive. “When it comes to the treatment of seizure clusters in pediatric patients with epilepsy five years of age and under, physicians and caregivers have limited options. We believe Libervant, as an oral alternative to existing device-based products, will be well-received by this patient population, if approved with market access.”
Libervant is a buccally, or inside of the cheek, administered film formulation of diazepam, a benzodiazepine intended for the acute treatment of intermittent, stereotypic episodes of frequent seizure activity (i.e., seizure clusters, acute repetitive seizures) that are distinct from a patient’s usual seizure pattern. Aquestive developed Libervant as an alternative to the device-based products currently available for patients with refractory epilepsy, including a rectal gel and nasal spray products. The FDA has granted tentative approval for Libervant for treatment of these epilepsy patients 12 years of age and older, with U.S. market access for Libervant for this age group of patients subject to the expiration of the existing orphan drug market exclusivity of a previously FDA approved drug scheduled to expire in January 2027. The NDA submitted today for Libervant for epilepsy patients between two and five years of age is subject to FDA approval, including for U.S. market access.
Aquestive is a pharmaceutical company advancing medicines to solve patients’ problems with current standards of care and provide transformative products to improve their lives. We are developing orally administered products to deliver complex molecules, providing novel alternatives to invasive and inconvenient standard of care therapies. Aquestive has five commercialized products marketed by its licensees in the U.S. and around the world, and is the exclusive manufacturer of these licensed products. The Company also collaborates with pharmaceutical companies to bring new molecules to market using proprietary, best-in-class technologies, like PharmFilm®, and has proven drug development and commercialization capabilities. Aquestive is advancing a late-stage proprietary product pipeline focused on treating diseases of the central nervous system and an earlier stage pipeline for the treatment of severe allergic reactions, including anaphylaxis.
prnewswire | August 29, 2023
Halozyme Therapeutics, Inc. announced the approval of Roche's Tecentriq® SC (atezolizumab), using Halozyme's ENHANZE® drug delivery technology, by the Medicines and Healthcare Products Regulatory Agency (MHRA) in Great Britain. Tecentriq® subcutaneous (SC) is co-formulated with Halozyme's proprietary recombinant human hyaluronidase enzyme, rHuPH20, allowing for subcutaneous delivery, which takes approximately seven minutes, compared with 30-60 minutes for intravenous (IV) infusion. Tecentriq® SC will be available to patients in Great Britain for certain types of lung, bladder, breast, and liver cancer.7
Multiple oncology studies suggest that most cancer patients prefer to receive their treatments via subcutaneous delivery, instead of intravenous infusion because of reduced discomfort, ease of administration, and shorter duration of treatment.2,4,11
"We are delighted that Roche has received approval of Tecentriq SC in Great Britain. Subcutaneous delivery of Tecentriq provides cancer patients a faster and more flexible treatment option and may alleviate pressure on healthcare system resources," said Dr. Helen Torley, president and chief executive officer of Halozyme. "We look forward to additional regulatory approvals of our partner products utilizing ENHANZE."
Tecentriq® SC is the first subcutaneous anti-PD-(L)1 cancer immunotherapy available to patients in Great Britain and is Roche's fourth subcutaneous cancer therapy using Halozyme's ENHANZE® drug delivery technology.8-10 The approval is based on clinical data from the Phase IB/III IMscin001 study, which showed comparable levels of Tecentriq® in the blood when administered subcutaneously, and a safety and efficacy profile consistent with the IV formulation.12 While the IMscin001 trial was conducted within the hospital setting, the SC formulation may be suitable for out-of-hospital administration by a healthcare professional.
The U.S. Food and Drug Administration (FDA) is currently evaluating Tecentriq® SC for regulatory approval.
Halozyme is a biopharmaceutical company bringing disruptive solutions to significantly improve patient experiences and outcomes for emerging and established therapies. As the innovators of the ENHANZE® technology with the proprietary enzyme rHuPH20, Halozyme's commercially-validated solution is used to facilitate the delivery of injected drugs and fluids in order to reduce the treatment burden to patients. Having touched more than 700,000 patient lives in post-marketing use in six commercialized products across more than 100 global markets, Halozyme has licensed its ENHANZE® technology to leading pharmaceutical and biotechnology companies including Roche, Takeda, Pfizer, AbbVie, Eli Lilly, Bristol-Myers Squibb, Alexion, argenx, Horizon Therapeutics, ViiV Healthcare and Chugai Pharmaceutical.
Halozyme also develops, manufactures and commercializes, for itself or with partners, drug-device combination products using its advanced auto-injector technology that are designed to provide commercial or functional advantages such as improved convenience and tolerability, and enhanced patient comfort and adherence. The Company has a commercial portfolio of proprietary products including XYOSTED®, TLANDO® and NOCDURNA® and partnered commercial products and ongoing product development programs with several pharmaceutical companies including Teva Pharmaceuticals and Idorsia Pharmaceuticals.
businesswire | August 25, 2023
Novaliq GmbH, a biopharmaceutical company focusing on first- and best-in-class ocular therapeutics, announced that the European Medicines Agency (EMA) has accepted the marketing authorization application (MAA) for CyclASol (ciclosporin ophthalmic solution) for regulatory review. CyclASol is an investigational new drug treatment for dry eye disease in patients not responding to tear substitutes. The Committee for Medicinal Products for Human Use (CHMP) of the EMA will start its review of the MAA under the centralized procedure for all 27 member states of the European Union (EU).
Dry eye disease (DED) affects millions of people in Europe and is one of the most common ocular surface disorders. A leading cause of DED is inflammation of the ocular surface. The chronic inflammatory nature causes progressive corneal surface damage that can lead to direct or indirect visual impairment. In the EU only one drug therapy limited to a subset of DED patients with severe keratitis is currently approved.
CyclASol is ciclosporin, solubilized in a novel, water-free excipient and was designed to address unmet needs, providing patients with a fast acting and well tolerable dry eye drug therapy. Dispensed in a unique 10 microliter small drop the solution does not contain water or anti-microbial preservatives, oils or surfactants. As a water-free product, there is no associated pH and no osmolarity.
“Our in Heidelberg, Germany, invented water-free technology was designed to unfold the full potential of pharmaceuticals on the ocular surface,” said Christian Roesky, Ph.D., Chief Executive Officer of Novaliq. “The acceptance of the EU Marketing Authorization Application is a key milestone in our global efforts to address high unmet needs to better serve patients suffering from dry eye disease. We look forward to collaborating with the CHMP throughout the review process and hope to make CyclASol available for patients in Europe.”
The clinical development program for the MAA is based on six clinical studies, which evaluated the safety and efficacy of CyclASol in a total of 1,575 patients with dry eye disease. Results from the two registrational studies, ESSENCE-1 and ESSENCE-2 consistently demonstrated that the product is effective for the treatment of DED. Continued improvement under therapy in both signs and symptoms of DED has been clinically demonstrated over a period of up to 56 weeks in an extension study of ESSENCE‑2. With its early onset of effect and its good tolerability profile CyclASol addresses unmet medical needs in DED.
CyclASol was approved by the U.S. Food and Drug Administration (FDA) as VEVYE™ (cyclosporine ophthalmic solution) 0.1% for the treatment of the signs and symptoms of dry eye disease on May 30, 2023. VEVYE is the first and only FDA approved cyclosporine solution indicated for the treatment of signs and symptoms of dry eye disease with efficacy demonstrated after 4 weeks of treatment and is commercialized in Northern America by Harrow Health Inc, a leading U.S. eyecare pharmaceutical company.
Novaliq is a private biopharmaceutical company focusing on the development and commercialization of first- and best-in-class ocular therapeutics based on EyeSol® the worldwide first water-free ophthalmic technology.
Novaliq offers an industry-leading portfolio addressing today's unmet medical needs of millions of patients with eye diseases based on its proprietary water-free eyedrop technology. On May 18th, 2023, U.S. Food and Drug Administration (FDA) approved MIEBO™ (perfluorohexyloctane ophthalmic solution; formerly known as NOV03) followed by the approval of VEVYE™ (cyclosporine ophthalmic solution, development name CyclASol®) 0.1% on May 30th, 2023.