Celnova Pharma Selected by Alnylam Pharmaceuticals for Exclusive Distribution Agreement in Colombia for GIVLAARI® (givosiran), the First-in-Class RNAi Therapeutic for the Treatment of Acute Hepatic Porphyria (AHP)
− GIVLAARI is the First Therapy Proven to Prevent AHP Attacks –
Bogota, Colombia, November 18th, 2020 – Celnova Pharma, a Latin American company focused in complex therapeutic areas, announced today that the company has entered into an exclusive distribution agreement with Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) for GIVLAARI® (givosiran), a first-in-class RNAi therapeutic for the treatment of acute hepatic porphyria (AHP).
“We are proud of our partnership with the worldwide leading RNAi therapeutics company,” said Augusto Penayo, General Manager of Celnova Pharma Colombia. “This encourages us to continue increasing awareness of this rare condition, providing education to physicians among different involved medical specialties.”
“Our partnership with Celnova enables us to extend access to GIVLAARI, pending INVIMA approval, to patients living with AHP in Colombia who are in urgent need of new treatment options,” said Norton Oliveira, Senior Vice President and Head of Latin America, Alnylam Pharmaceuticals. “AHP can cause potentially life-threatening attacks and for some patients, chronic, painful symptoms. We are proud to provide a long-sought treatment option that can help with the debilitating manifestations of AHP and unpredictable nature of AHP attacks. We look forward to working with INVIMA to assure the fastest possible regulatory approval.”
About GIVLAARI® (givosiran)
GIVLAARI is an RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) approved for the treatment of adults with acute hepatic porphyria (AHP) in the U.S., Brazil and Canada, and for the treatment of AHP in adults and adolescents aged 12 years and older in the EU. In the pivotal ENVISION Phase 3 study, givosiran was shown to significantly reduce the annualized rate of composite porphyria attacks that required hospitalization, urgent healthcare visit or intravenous hemin administration at home compared to placebo. GIVLAARI is Alnylam’s first commercially available therapeutic based on its Enhanced Stabilization Chemistry ESC-GalNAc conjugate technology to increase potency and durability. GIVLAARI is administered via subcutaneous injection once monthly at a dose based on actual body weight and should be administered by a healthcare professional. GIVLAARI works by specifically reducing elevated levels of ALAS1 messenger RNA, leading to reduction of toxins associated with attacks and other disease manifestations of AHP.
About Acute Hepatic Porphyria
Acute hepatic porphyria (AHP) refers to a family of ultra-rare, genetic diseases characterized by debilitating, potentially life-threatening attacks and, for some patients, chronic manifestations that negatively impact daily functioning and quality of life. AHP is comprised of four subtypes: acute intermittent porphyria (AIP), hereditary coproporphyria (HCP), variegate porphyria (VP), and ALA dehydratase-deficiency porphyria (ADP). Each type of AHP results from a genetic defect leading to a lack of certain enzymes needed to produce heme in the liver, which leads to an accumulation of porphyrins in the body to toxic amounts. AHP disproportionately impacts women of working and childbearing age, and symptoms of the disease vary widely. Severe, unexplained abdominal pain is the most common symptom, which can be accompanied by limb, back, or chest pain, nausea, vomiting, confusion, anxiety, seizures, weak limbs, constipation, diarrhea, or dark or reddish urine. AHP is life-threatening due to the possibility of paralysis and respiratory arrest during attacks. The nonspecific nature of AHP signs and symptoms can often lead to misdiagnoses of other more common conditions such as gynecological disorders, viral gastroenteritis, irritable bowel syndrome (IBS), and appendicitis. Consequently, on a global perspective, patients with AHP can wait up to 15 years for a confirmed diagnosis, with the risk of addiction problems. In addition, long-term complications and comorbidities of AHP can include hypertension, chronic kidney disease or liver disease, including hepatocellular carcinoma.
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.
Alnylam (Nasdaq: ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform. Alnylam’s commercial RNAi therapeutic products are ONPATTRO® (patisiran), approved in the U.S., EU, Canada, Japan, Brazil, and Switzerland, and GIVLAARI® (givosiran), approved in the U.S., EU, Brazil, and Canada. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its “Alnylam 2020” strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines to address the needs of patients who have limited or inadequate treatment options and now expects to exceed its “Alnylam 2020” guidance. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam or on LinkedIn.
About Celnova Pharma
Celnova Pharma is a pharmaceutical company focused in complex therapeutic classes, with a strong presence in oncology, central nervous system and rare diseases. Through its work in Latin America, Celnova aims to preserve and improve the lives of people facing serious medical conditions.
Celnova’s business model combines proprietary branded generics with in-licensing of original and difficult-to-make generic drugs. Its long term goal is to grow in Latin America through a mix of organic growth and selected acquisitions and partnerships
With direct presence in Argentina, Chile, Peru and Colombia, Celnova commercializes a portfolio of products through its own sales force, reaching specialist doctors and institutional buyers.
For more information about Celnova Pharma, visit www.celnova.com.