Here are below some recent 505b2 approvals with value additions and different approaches to address unmet need of patients.

 

1. XROMI (Hydroxyurea), Oral solution
Approval: Apr 04, 2024
Applicant: Nova Labs
Indication: sickle cell anemia with recurrent moderate to severe painful crises in pediatric patients aged 6 months of age to less than 2 years
PREA: Exempted because of orphan drug designation
Value addition: First oral liquid formulation for pediatric patients

 

2. RETEVMO (Selpercatinib), tablet
Approval: Apr. 10, 2024
Applicant: Loxo Onco / Eli Lilly
Indication: Non-Small Cell Lung Cancer; Thyroid Cancer; Solid tumors
PREA: Exempted because of orphan drug designation
Value addition: Tablet dosage forms with additional strengths

 

3. ENTRESTO SPRINKLE (Sacubitril + Valsartan), pellets capsules
Approval: Apr 12, 2024
Applicant: Novartis
Indication: Adult & Pediatric Heart Failure
PREA: Fulfilled pediatric study requirements for ages 1 month to less than 18 years of age. Waived for birth to less than 1 month of age because studies are impossible or highly impractical.
Value addition: Pediatric friendly formulation

 

4. REZENOPY (Naloxone HCl), Nasal Spray
Approval: Apr. 19, 2024
Applicant: Summit Bioscience
Indication: Emergency treatment of known or suspected opioid overdose
PREA: Not required. This product is appropriately labelled for use in all relevant pediatric populations.
Value addition: Rezenopy is a high dose formulation containing 10 mg of naloxone per spray.

 

5. LUMIFY PRESERVATIVE FREE (Brimonidine Tartarate), Ophthalmic drops
Approval: Apr 19, 2024
Applicant: Bausch & Lamb
Indication: Relief of redness of the eye due to minor eye irritations
PREA: Exempted because none of the PREA criteria apply
Value addition: Preservative free OTC product

 

6. PIVYA (Pivmecillinam), Tablet
Approval: Apr 24, 2024
Applicant: Utility Therapeutics Ltd
Indication: Uncomplicated urinary tract infection in female patients 18 years of age and older
PREA: Deferred as PMR for 2 months to less than 18 years. Waived for birth to less than 2 months as studies are impossible.
Value addition: Prodrug of mecillinam. Due to its unique MOA & stability against β-lactamase, it may address antibiotic resistance issues.

 

7. VIJOICE (Alpelisib), Oral granules
Approval: Apr 24, 2024
Applicant: Novartis
Indication: adult and pediatric patients 2 years of age and older with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS)
PREA: Exempted, because of an orphan drug designation
Value addition: Pediatric friendly formulation

 

8. RINVOQ LQ (Upadacitinib), Oral solution
Approval: Apr 26, 2024
Applicant: Abbvie
Indication: Patients 2 years of age and older with active polyarticular juvenile idiopathic arthritis & psoriatic arthritis
PREA: Exempted, because of an orphan drug designation
Value addition: Pediatric friendly formulation

 

9. LIBERVANT (Diazepam), Buccal film
Approval: Apr 26, 2024
Applicant: Aquestive Therapeutics
Indication: Intermittent, stereotypic episodes of frequent seizure activity in patients with epilepsy 2 to 5 years of age
PREA: Exempted, because of an orphan drug designation
Value addition: First and only FDA approved orally administered rescue product for the treatment of seizure clusters in patients ages 2 to 5. It provides a new way to deliver diazepam.