FDA grants Orphan Designation to Bausch’ Rifaximin for SCD

“Early data suggests that rifaximin may be beneficial in reducing vaso-occlusive crisis in patients with sickle cell disease, and we are currently finalizing trial protocols to further evaluate its potential in this patient population,” said Joseph C. Papa, chairman and CEO, Bausch Health. “We expect to start a Phase 2 trial in the first half of 2021 evaluating a novel rifaximin formulation for a potential sickle cell disease treatment, and we are hopeful to be able to bring this investigational oral formulation forward to help sickle cell patients.” 

Approximately 100,000 Americans have sickle cell disease, which is a group of inherited red blood cell disorders that affects hemoglobin, the protein that carries oxygen through the body. Patients with sickle cell disease have crescent or “sickle” shaped red blood cells that can block blood flow through the body, which can lead to serious problems, including stroke, eye problems, infections and unpredicted episodes of vaso-occlusive crisis, which is described as sharp, intense, stabbing or throbbing pain that can strike nearly anywhere in the body and often requires intravenous pain medication. Sickle cell disease is a lifelong illness, and its only cure is a blood and bone marrow transplant.1

FDA priority review of Regeneron sBLA for Libtayo in NSCLC

The sBLA is supported by results from a Phase 3 open-label, randomized, multi-center trial that investigated the first-line treatment of Libtayo monotherapy compared to platinum-doublet chemotherapy in patients with locally advanced or metastatic NSCLC whose tumor cells expressed PD-L1, including those whose cancers had confirmed PD-L1 expression of ≥50% using the PD-L1 IHC 22C3 pharmDx kit. Results were recently presented at the 2020 European Society for Medical Oncology (ESMO) Virtual Congress in September.

The European Medicines Agency (EMA) is also assessing Libtayo in advanced NSCLC with ≥50% PD-L1 expression and a decision is expected in the second quarter of 2021. Libtayo is the first systemic treatment approved in the U.S. and European Union (EU) for adults with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation.

Libtayo is being jointly developed and commercialized by Regeneron and Sanofi under a global collaboration agreement. The use of Libtayo to treat advanced NSCLC is investigational and has not been fully evaluated by any regulatory authority.

Libtayo is a fully-human monoclonal antibody targeting the immune checkpoint receptor PD-1 on T-cells. By binding to PD-1, Libtayo has been shown to block cancer cells from using the PD-1 pathway to suppress T-cell activation.

Lung cancer is the leading cause of cancer death worldwide, with more than 2.2 million new cases expected globally, and 228,000 new cases expected in the U.S. in 2020. Approximately 85% of all lung cancers are NSCLC, and an estimated 25% to 30% of these cases are expected to test positive for PD-L1 in ≥50% of tumor cells. While immunotherapies have transformed advanced NSCLC treatment in recent years, there remains an unmet need to optimize the identification and treatment of patients with high PD-L1 expression and offer additional treatment options.

Sanofi THOR-707 and Merck Keytruda to be evaluated for cancer combotherapy

Under the agreement, Sanofi will sponsor the clinical trials while MSD will provide KEYTRUDA. 

THOR-707 is currently being evaluated by Sanofi in an ongoing Phase 1 open-label, multi-center, dose escalation and expansion trial.  This study is designed to evaluate the safety and tolerability of THOR-707, and to determine its recommended Phase 2 dose alone and in combination with anti-PD-1 and anti-EGFR antibodies. 

In addition to testing THOR-707 in combination with KEYTRUDA, Sanofi is separately evaluating the activity of this novel biologic in combination with other anti-PD-1 antibodies, including Libtayo® (cemiplimab-rwlc) and with anti-EGFR and anti-CD38 antibodies for various types of cancer tumors. 

THOR-707 demonstrated in preclinical studies the ability to induce the expansion of CD8+T-cells resulting in anti-tumor effects both as single agent as well as in combination with an anti-PD1 mAb.

It is the first molecule from the Synthorin platform, Sanofi’s unique expanded genetic alphabet platform, which has the potential to create a new generation of precision medicines for oncology and autoimmune disease. 

EC approves Insmed’s ARIKAYCE for NTM lung infections caused by MAC

The EC approval of ARIKAYCE is based on results from the randomized, open-label, global Phase 3 CONVERT study, which demonstrated that once-daily ARIKAYCE, when combined with multi-drug regimen (MDR), improved sputum culture conversion rates in patients with refractory NTM lung disease caused by MAC compared to MDR therapy alone. The most common side effects with ARIKAYCE affecting the respiratory system are dysphonia, cough, dyspnea, and hemoptysis.

The EC approval follows a positive opinion from the Committee for Medicinal Products for Human Use of the European Medicines Agency (EMA) on July 24, 2020. In addition, the Committee for Orphan Medicinal Products of the EMA has adopted a positive opinion recommending that ARIKAYCE maintain Orphan Drug Designation in the EU for the treatment of NTM lung disease, which was originally granted to Insmed in 2014. 

Insmed plans to launch ARIKAYCE first in Germany, followed by the United Kingdom (UK) and other EU markets, subject to local reimbursement processes. As part of Insmed’s comprehensive approach to patient support, the Company has established country-specific programs to provide patients with direct and ongoing support and information.

In the U.S., ARIKAYCE (under the generic name amikacin liposome inhalation suspension), is the first and only approved treatment for MAC lung disease as part of a combination antibacterial drug regimen for adult patients with limited or no alternative treatment options. Insmed has submitted a new drug application for ARIKAYCE in Japan for the treatment of patients with NTM lung disease caused by MAC who did not sufficiently respond to prior treatment.

Mycobacterium avium complex (MAC) lung disease is a rare and serious disorder that can significantly increase morbidity and mortality. Patients with MAC lung disease can experience a range of symptoms that often worsen over time, including chronic cough, dyspnea, fatigue, fever, weight loss, and chest pain. In some cases, MAC lung disease can cause severe, even permanent damage to the lungs, and can be fatal. MAC lung disease is an emerging public health concern worldwide with significant unmet need.

Orphan Drug and Rare Pediatric status for Taysha’s TSHA-104 in SURF1-associated Leigh syndrome

Taysha has secured rare pediatric disease designation and orphan drug designation for multiple programs, including GM2 gangliosidosis (-101), CLN1 (-118), Rett syndrome (-102) and now SURF1 (-104). In addition to these designations, the company also has fast track status for the CLN1 program. 

Leigh syndrome is a severe neurological disorder that usually presents in the first year of life. It is characterized by progressive loss of mental and movement abilities that can result in death within two to three years. Approximately 10-15% of people with Leigh syndrome have a SURF1 mutation.