The DELIVER results indicate that AstraZeneca is on course to regain parity with its SGLT2 drug in Heart Failure, based on earlier clinical results in HFpEF in the phase 2 PRESERVED-HF trial. Meanwhile, Novartis’ Entresto (sacubitril/valsartan) has been approved in the US for both HFrEF and HFpEF, but its labeling in the EU has not been expanded beyond HFrEF. Overall, the evidence for Entresto in HFpEF appears to be poorer than for Jardiance, while there is a possibility for combination use due to the different modes of action. Moreover, with the approval of Farxiga in Chronic Kidney Disease (CKD), AstraZeneca has the first-mover advantage in another potentially profitable market for SGLT2 drugs. Whereas Boehringer and Lilly, on the other hand, have just announced Jardiance’s first-in-class success in Acute Heart Failure (AHF).
EU Approves Incyte’s Jakavi for Acute and Chronic Graft-Versus-Host Disease
The European Commission (EC) has approved Jakavi (ruxolitinib) for the treatment of patients aged 12 years and older with acute or chronic GvHD who have not responded to corticosteroids or other systemic treatments. The approval of Jakavi follows a positive opinion issued in March by the European Medicines Agency’s Committee for Medicinal Products for Human Use, which was based on the Phase III REACH2 and REACH3 trials, in which Jakavi demonstrated superiority in overall response rate (ORR) compared to best available therapy (BAT). REACH2 results indicated a 62% ORR with Jakavi at Day 28, compared to 39% for BAT; and REACH3 results showed a considerably improved ORR at week 24 (50% vs. 26%) with a higher best ORR (76% vs. 60%) vs. BAT, among steroid-refractory/dependent chronic GvHD patients. The approval of Jakavi provides healthcare practitioners and individuals with GvHD who are still dependent on or resistant to steroids with a new option for managing this debilitating and potentially fatal condition.
Incyte licensed ruxolitinib to Novartis for development and commercialization outside the US. Incyte markets ruxolitinib as Jakafi® in the United States for adults with Polycythemia Vera who have had an inadequate response to or are intolerant of hydroxyurea, adults with intermediate or high-risk MF, adult and pediatric patients 12 years and older with steroid-refractory acute GvHD, and adult and pediatric patients 12 years and older with chronic GvHD after the failure of one or two lines of corticosteroids or other systemic therapy.
Idorsia Launched its Insomnia Drug Quviviq in the Market
Idorsia Ltd & Idorsia Pharmaceuticals has launched QUVIVIQ™ (daridorexant) CIV 25 mg and 50 mg tablets in the US for adult patients with insomnia. The therapy QUVIVIQ™ is intended for patients having trouble with falling or staying asleep. With the launch of the therapy, QUVIVIQ becomes Idorsia’s first commercial product in the United States.
QUVIVIQ is a dual orexin receptor antagonist, which blocks the binding of the wake-promoting neuropeptides called orexins in the brain and is thought to turn down overactive wakefulness in insomnia. QUVIVIQ is available as 25-mg and 50-mg tablets and is recommended once per night, taken orally within 30 minutes before going to bed, with at least seven hours remaining prior to planned awakening.
Idorsia received the US Food and Drug Administration (FDA) for QUVIVIQ™ (daridorexant) in January 2022. The approval was based on the extensive clinical program that included 1,854 adults with insomnia at over 160 clinical trial sites across 18 countries.
Insomnia is the most common sleep disorder and affects nearly 25 million adults in the US. It is associated with numerous serious health conditions, such as psychiatric disorders, cardiovascular disease, substance abuse, and dementia. Insomnia is usually a temporary or short-term condition. However, in some cases, insomnia can become chronic or long-term, if the patient experiences difficulties in sleeping at least three days a week for at least a month. As per DelveInsight, in 2020, Chronic Insomnia prevalent cases in the US were found to be 1,763,000 and 811,000 for females and males respectively which is expected to increase in the coming years. The launch of the therapy is expected to immensely improve the health outcome of the patient affected with insomnia.
FDA Grants Fast Track Status to HM43239 for R/R Acute Myeloid Leukemia
Aptose Biosciences, a clinical-stage precision oncology company has announced that the FDA has granted Fast Track designation to HM43239. HM43239 is an oral, myeloid kinome inhibitor, for the treatment of patients with relapsed or refractory (R/R) Acute Myeloid Leukemia (AML) with FLT3 mutation. The decision is based on the ongoing phase 1/2 trial (NCT03850574) in which the drug-induced complete remissions (CRs) continue to be a well-tolerated oral agent. Earlier in 2018, FDA granted orphan drug designation to HM43239 for the treatment of acute myeloid leukemia.
Currently, an international Phase 1/2 clinical trial is underway for HM43239 in the R/R AML. The trial was designed to evaluate the pharmacodynamic response, pharmacokinetics, safety, and tolerability of the drug as a single agent. HM43239 has produced complete remission in a diversity of individuals with R/R AML.
As per DelveInsight, Acute Myeloid Leukemia (AML) is one of the most common types of leukemia in adults and primarily affects older people with a median age of diagnosis of 67 years. AML accounts for nearly 1% of all cancers and is found to be more prominent in males in comparison to females. The American Cancer Society estimates there will be 60,650 new cases of leukemia diagnosed in 2022, with 20,050 being new cases of AML, mostly in adults.
EU Approves Kymirah, First CAR-T Therapy For Follicular Lymphoma
Novartis declared today that the European Commission had approved Kymriah®, a CAR-T cell therapy, to treat adult patients suffering from relapsed or refractory follicular lymphoma after two or more lines of systemic therapy. The approval follows an affirmative opinion in March by the Committee for Medicinal Products for Human Use of the European Medicines Agency. This approval declares the third indication for Kymriah, making it the first CAR-T cell therapy approved in the European Union for these patients, including those with relapsed/refractory follicular lymphoma grades 1, 2, and 3A.
The approval is established on the global Phase II ELARA trial, demonstrating that 86% of patients treated with Kymriah had a response, including 69% of patients who had a complete response. Lengthy durable response to treatment was shown, with an estimated 87% of patients who achieved a complete response still in response at or more than nine months after initial response. In the trial, it was observed that 94 infused patients were assessed for efficacy with a follow-up period of around 21 months.
Among 97 patients evaluable for safety, the safety profile of Kymriah was remarkable. Cytokine release syndrome was reported in 50% of patients after Kymriah infusion, and no Grade 3 or 4 events were reported, as defined by the Lee scale.
Neurological adverse reactions occurred in 9% of patients within eight weeks after Kymriah infusion. Severe infections occurred in 16% of patients.
In addition to relapsed/refractory Follicular Lymphoma, Kymriah is approved for the treatment of pediatric and young adult patients upto and including 25 years of age with B-cell acute lymphoblastic leukemia that is refractory/in relapse post-transplant/in second or later relapse, and adult patients with relapsed/refractory diffuse large B cell lymphoma after two or more lines of systemic therapy.
Perrigo Completes the Acquisition of HRA Pharma
Perrigo Company plc (NYSE: PRGO), located in Dublin, stated that on April 29, 2022, it completed the previously announced acquisition of Héra SAS, better known as HRA Pharma, situated in Châtillon, France, for approximately €1.8 billion or $1.9 billion in cash (based on current exchange rates).
HRA Pharma, founded in 1996, creates over-the-counter and consumer health care products. The company specializes in women’s health and advertises itself as a European leader in emergency contraception and skin problems, with “revolutionary Rx-to-OTC switches” in the pipeline. HRA Pharma has direct operations in 11 European countries and has created agreements that have allowed it to sell its products in more than 71 countries across the world.
Perrigo Company plc, on the other hand, is a major developer of Consumer Self-Care Products and over-the-counter (OTC) health and wellness products that help individuals prevent or treat self-managed conditions. Perrigo is the largest store brand OTC player in the United States competing, with more than 9,000 SKUs under customer ‘own brand’ labels, thanks to its consumer self-care approach. Perrigo is also a Top 10 OTC firm in Europe by revenue, with over 200 branded OTC products distributed across 28 countries.
HRA’s acquisition furthers Perrigo’s goal of improving lives with high-quality, low-cost self-care products that customers can trust wherever they are marketed. It also increases sales and profit growth and is predicted to improve the company’s margins. The multinational businesses are more interconnected and united than ever before, and Perrigo’s consumer-centric philosophy is being leveraged as the focus switches to growing regional reach, driving category penetration, and providing new product innovation.
Janssen Submits Marketing Authorisation Application to EMA Seeking Approval of Niraparib and Abirateron
The Janssen Pharmaceutical Companies of Johnson & Johnson recently announced the submission of a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for approval of niraparib in combination with abiraterone acetate in the form of a dual action tablet (DAT) along with prednisolone for the treatment of patients with Metastatic Castration-Resistant Prostate Cancer (mCRPC) who are positive for homologous recombination repair (HRR)+ gene alterations. Niraparib in conjunction with AAP will be the first dual action tablet formulation in the European Union to precisely target HRR gene changes in mCRPC if authorised by the European Commission.
In patients with mCRPC, the combination of the niraparib, a PARP (poly adenosine diphosphate-ribose polymerase) inhibitor and the CYP17 inhibitor abiraterone acetate targets two oncogenic drivers, the AR-axis and HRR gene mutations. The DAT formulation also attempts to increase treatment compliance by making it more comfortable for patients.
The EU MAA is based on results from the MAGNITUDE trial (NCT03748641), a Phase 3 randomized, double-blind, placebo-controlled, multicenter research that investigated at the safety and efficacy of niraparib in combination with abiraterone acetate plus prednisone (AAP) in patients with mCRPC. The therapeutic combination of niraparib and AAP exhibited a statistically significant improvement in individuals with HRR gene mutations as compared to placebo and AAP in the final analysis for radiographic progression-free survival (rPFS).
With roughly 473,000 people diagnosed in 2020, Prostate Cancer is one of the most prevalent cancers in Europe. HRR gene mutations are seen in up to 30% of individuals with mCRPC and are linked to a poor outcome when compared to those who do not have HRR gene mutations. The European Medicines Agency’s approval of niraparib in conjunction with AAP represents a significant step forward in treating a particular genetic alteration in prostate cancer.
