Pharmaceutical Technology Editors

Longeveron, a clinical stage biotechnology company, announced on Dec. 6, 201, that FDA granted Orphan Drug Designation (ODD) to Lomecel-B, an allogenic injection designed to treat age-related chronic illnesses. The ODD was granted for the treatment of Hypoplastic Left Heart Syndrome (HLHS), a rare and life-threatening congenital heart defect in infants.

HLHS is a condition wherein the left ventricle is underdeveloped, resulting in impaired ability to pump blood through the body; it is often fatal. According to a company press release, HLHS occurs in approximately 1000 babies in the United States each year. Because the incidence rate is this low, it qualifies for an ODD, which grants exclusive benefits to treatments designed for rare and underserved conditions.

“Adding to the Rare Pediatric Disease designation already granted to Lomecel-B for treatment of HLHS, the FDA’s decision to grant ODD to Lomecel-B for this indication indicates the ongoing and unmet need for new therapies to treat infants with HLHS,” said Geoff Green, CEO at Longeveron, in the press release. “Building on results from our completed Phase 1 safety-focused trial, we believe Lomecel-B has potential to improve outcomes for these severely impacted infants by way of repairing cardiac tissue and improving ventricular function.”

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Longeveron’s Lomacel-B received an Orphan Drug Designation for the treatment of Hypoplastic Left Heart Syndrome.

FDA Grants Longeveron Orphan Drug Designation for Lomacel-B

Salubrent announced that they had filled two positions at the company’s recently opened cGMP analytical laboratory in Kannapolis, NC on Dec. 7, 2021. Annad Padmanabhan will join as Director of Analytical Development, while Maggie Thomasson will fill the role of Analytical Development Chemist.

Padmanabhan brings more than 29 years of experience in analytical R&D and quality control; in particular, he specializes in analytical method development and method validation. He has previously worked with Glenmark Pharmaceuticals, G&W Laboratories, Ei LLC, and Applied Analytical Industries, among others.

Thomasson brings more than five years of method development and validation experience with high-performance liquid chromatography, mass spectrometry, nuclear magnetic resonance, cell culture/lysis, and protein expression. Thomasson has a deep understanding of many safety and regulatory compliance guidelines. She has worked with Sentia Wellness and Pfizer (Aerotek).

“Adding these two seasoned experts to our team is representative of our commitment to providing the Salubrent customer experience to customers in need of comprehensive analytical testing services, including stability program management,” said Terry Novak, president & CEO of Salubrent Pharma Solutions, in a company press release. “The new hires allow Salubrent to fully utilize its new analytical lab and further extend the expertise, experience, and capabilities that we bring to our customers.”

Annad Padmanabhan and Maggie Thomasson will fill key roles at Salubrent’s new cGMP analytical laboratory in Kannapolis, NC.

Salubrent Pharma Solutions Strengthens Analytical Team

Recordati acquired EUSA Pharma, a pharmaceutical company specializing in developing oncology treatments, for $845 million on Dec. 3, 2021. This acquisition grants Recordati access to EUSA’s portfolio of rare cancer treatments.

Included in the portfolio are four treatments:

 is an anti-GD2 monoclonal antibody (mAb) designed for the treatment of high-risk neuroblastoma. According to a company press release, neuroblastoma is a rare form of cancer that forms as a result of immature nerve cells and predominantly affects children under the age of ten. Dinutuximab beta is currently approved in Europe and other countries

 is an anti-IL-6 mAb designed for the treatment of Idiopathic multicentric Castleman’s disease (iMCD). iMCD is a variation of Castleman’s disease, a rare lymph node cancer, that results in severe systemic symptoms. Siltuximab is the first and only approved treatment for iMCD in the United States and Europe

 is an oral small molecule tyrosine kinase inhibitor of vascular endothelial growth factor receptors 1, 2, and 3. It is currently approved for first-line treatment of advanced renal cell carcinoma in Europe and other countries

 is an oral solution for the treatment of oral mucositis resulting from chemo- and radiotherapy. It is currently approved in the US, EU, and other markets.

“We believe that the EUSA Pharma acquisition represents an excellent opportunity to further expand and reinforce our portfolio in a new and underserved therapeutic area, rare and niche oncology, with high potential growth products and will provide a platform for potential further future expansion in these areas,” said Andrea Recordati, chairman, Recordati, in a company press release. “This is another important step forward in delivering on our strategy to increase our presence in the rare disease segment and to fulfil our mission to improve the lives of patients, delivering innovative treatments that address serious unmet medical needs.”

Recordati’s blockbuster acquisition of EUSA Pharma gives them access to a portfolio of four drugs that treat rare cancers.

Recordati Acquires EUSA Pharma for $845 Million

The Antimicrobial Drugs Advisory Committee (AMDAC) voted on Nov. 30, 2021, to recommend an Emergency Use Authorization (EUA) for molnupiravir, an experimental antiviral oral COVID-19 medication from Merck—known as MSD outside of the United States and Canada—and Ridgeback Biotherapeutics. While questions about its risks cropped up following initial positive findings, a 13-10 decision was ultimately made in favor of its recommendation.

Merck announced positive preliminary results for molnupiravir on Oct. 1st, 2021. 

, molnupiravir reduced the risk of hospitalization or death by 50% in non-hospitalized adult COVID-19 patients with mild-to-moderate symptoms. Following these results, Merck was advised to end the trial early and applied for an EUA on Oct. 8, 2021.

However, following an evaluation of the full population, this risk reduction was instead found to be approximately 30%. This reduced effectivity, in tandem with concerns about potential risks to fetuses and DNA, led to many members of the panel to believe the potential risk was not worth the demonstrated benefit.

While FDA is not required to follow the recommendations of AMDAC, they typically do. Should molnupiravir be approved, it would become the first oral treatment for COVID-19.

Following mixed results from clinical trials, a panel of outside experts voted 13-10 to recommend Merck’s pill for Emergency Use Authorization.

FDA Panel Recommends Merck’s COVID-19 Pill 

FDA approved Genentech’s rituximab (Rituxan) in combination with chemotherapy for pediatric patients (6 months of age and older to 18 years of age) with previously untreated, advanced stage, CD20-positive diffuse large B-cell lymphoma (DLBCL), Burkitt lymphoma (BL), Burkitt-like lymphoma (BLL), or mature B-cell acute leukemia (B-AL), according to a Dec. 2, 2021, press release.

When given as an intravenous infusion given in combination with systemic Lymphome Malin B (LMB) chemotherapy, the recommended rituximab dose is 375 mg/m

. Six total infusions of rituximab are given.

A global multicenter, open-label, randomized trial was performed, assessing patients six months in age or older with previously untreated, advanced stage, CD20-positive DLBCL/BL/BLL/B-AL. Event-free survival was the main efficacy outcome measure. This was defined as a progressive disease, relapse, second malignancy, death from any cause, or non-response as evidenced by detection of viable cells in residue after the second Cytarabine Aracytine, Ara-C, Veposide course. There were 20 deaths in the LMB chemotherapy arm compared to 8 deaths in the rituximab plus LMB chemotherapy arm at the time of the interim analysis.

Subtitle FDA approved rituximab in combination with chemotherapy for pediatric patients with previously untreated, advanced stage, CD20-positive DLBCL/BL/BLL/B-AL.

Rituximab Plus Chemotherapy Approved by FDA for Select Pediatric Cancers

On Dec. 1, 2021, Roche announced the completion of its share purchase agreement to acquire 100% of the outstanding shares of TIB Molbiol Group in a company press release. The companies will focus on the rapid development of polymerase chain reaction (PCR) assays and diagnostic tests for emerging pathogens and potential health threats, such as infectious diseases. TIB Molbiol will operate as a subsidiary within the Diagnostics division.

For more than 20 years, the companies have collaborated to address biological threats, including:

“I am very pleased that together with TIB Molbiol, we can significantly improve access for patients to a broader spectrum of diagnostic tests—from high volume routine tests to the diagnosis of rare diseases. TIB Molbiol will continue to be an innovation engine and frontrunner in our common fight against infectious diseases, such as SARS-CoV-2,” said Thomas Schinecker, CEO, Roche Diagnostics, in a company press release. “TIB Molbiol has helped to shape the future of PCR infectious disease testing and will continue to drive cutting-edge research so we can bring better healthcare outcomes to patients faster.”

The companies will focus on the rapid development of polymerase chain reaction assays for emerging pathogens and potential health threats, including biological threats.