Insights+: The US FDA New Drug Approvals in August 2022

• The US FDA approved 4 NDAs and 1 BLA in August 2022, leading to treatments for patients and advances in the healthcare industry. The CDER and CBER approved 47 novel products in 2022
• In August 2022, the major highlights drugs were Calquence’s approval for blood cancer, Imbruvica (ibrutinib) for chronic graft versus host disease in pediatric patients, Xenpozyme (olipudase alfa-rpcp) for acid sphingomyelinase deficiency
• PharmaShots has compiled a list of a total of 5 new drugs approved by the US FDA in August 2022

AstraZeneca’s Calquence (acalabrutinib) Receives the US FDA’s Approval for the Treatment of Blood Cancers

Calquence

Active ingredient: acalabrutinib                                                         Approved: August 1, 2022

Company: AstraZeneca                                                                       Disease: Blood Cancer

• The US FDA has approved Calquence as a new tablet formulation for all current indications incl. CLL, SLL & r/r MCL
• The approval was based on the (ELEVATE-PLUS) trials evaluating Calquence in 116 healthy patients which showed that acalabrutinib 100mg tablets were bioequivalent to the currently marketed acalabrutinib 100mg capsules indicating the same efficacy & safety profile with the same dosing strength. The tablet can be taken with gastric acid-reducing agents, incl. PPIs, antacids & H2-receptor antagonists & no new safety signals were reported
• Calquence is also approved as a capsule formulation for the same indications & as the tablet in the US and other countries globally

Axsome’s Auvelity Receives the US FDA’s Approval for the Treatment of Major Depressive Disorder

Auvelity

Active ingredient: dextromethorphan HBr -bupropion HCl             Approved: August 19, 2022

Company: Axsome Therapeutics                                                       Disease: Blood Cancer

• The US FDA has approved Auvelity (dextromethorphan HBr -bupropion HCl) ER tablets for MDD. The therapy is expected to be available in the US in the Q4’22
• The approval was based on the P-III (GEMINI) trial to assess Auvelity in 327 patients which showed significant superiority in improving symptoms of depression over PBO, change in MADRS total score from baseline was significant at 1 & 2wk.
• In (ASCEND) study, the therapy was superior to bupropion sustained-release tablets (105mg, BID). The company launched the patient support program i.e., Auvelity On My Side which provides access to patients to use Auvelity & also offers patient support services, incl. the Auvelity On My Side savings card for eligible commercially-insured patients

AbbVie’s Imbruvica (ibrutinib) Receives the US FDA’s Approval for Chronic Graft Versus Host Disease in Pediatric Patients Aged ≥1 Year

Imbruvica

Active ingredient: ibrutinib                                                                Approved: August 25, 2022

Company: AbbVie                                                                               Disease: Chronic Graft Versus Host Disease

• The US FDA has approved Imbruvica for cGVHD in pediatric patients aged ≥1yr. who required additional therapy after failure of ≥1 line of systemic therapy. The therapy is jointly developed & commercialized by Janssen & Pharmacyclics
• The approval was based on the P-I/II (iMAGINE) trial evaluating Imbruvica (in 47 pediatric & young adult patients aged 1yr. to ≤22yrs. with cGVHD which showed ORR (60%) @25wk. in patients aged 13yrs., m-DoR was 5.3mos. The safety was consistent with the established profile for Imbruvica, ARs consistent with those observed in cGVHD
• Imbruvica is a BTK inhibitor & was approved for a pediatric patient population. The therapy marks the first approved treatment option for children ≤12yrs. with cGVHD

Azurity’s Konvomep Receives the US FDA’s Approval for the Treatment of Active Benign Gastric Ulcer

Konvomep

Active ingredient: omeprazole and sodium bicarbonate                 Approved: August 31, 2022

Company: Azurity Pharmaceuticals                                                   Disease: Benign Gastric Ulcer

• The US FDA has approved Konvomep (omeprazole and sodium bicarbonate for oral suspension) for the treatment of active benign gastric. The therapy is also indicated for lowering the upper gastrointestinal bleeding risk in critically ill patients

• Konvomep is expected to be commercially available in Q1’23 & may give patients who have difficulty swallowing pills or capsules. Konvomep contains omeprazole (2mg), a proton pump inhibitor, and sodium bicarbonate per mL (84mg)
• The product is available in 90/150/300mL bottles. In July 2022, Azurity’s Zonisade has been approved in the US for partial seizures in adults and pediatric epilepsy patients aged ≥16yrs.

Sanofi’s Xenpozyme (olipudase alfa-rpcp) Receives the US FDA’s Approval as First Disease-Specific Treatment for Acid Sphingomyelinase Deficiency

Xenpozyme

Active ingredient: olipudase alfa-rpcp                                                Approved: August 31, 2022

Company: Sanofi                                                                                   Disease: Acid Sphingomyelinase Deficiency

• The approval was based on the (ASCEND) & (ASCEND-Peds) trials that evaluated Xenpozyme in 31 adult & 8 pediatric patients with ASMD (non-CNS manifestations) type A/B or type B for 52 & 64wks. Xenpozyme is expected to be available in the US in the coming wks.
• The results showed a mean reduction in spleen volume by (38.9%) from baseline to 52wk. over mean increase by 0.5% for patients in the PBO group in (ASCEND) trial & 46.7% in (ASCEND-Peds) trials; mean reduction in liver volume (26.5% vs 1.8%) & 38.1%; mean improvement in platelet count (18.3% vs 2.7%) & 37.6%
• In (ASCEND-Peds) trials, patients who performed the test at baseline had a mean relative improvement of 45.9% predicted DLco from baseline (48.5%) to 52wk. (70.9%) along with an improvement in lung function in both trials

Related Post: Insights+: The US FDA New Drug Approvals in July 2022