In this episode, we discuss a new formulation of treatment for HAE, the FDA approval of Voceryq for recurrent respiratory papillomatosis, and 3D printed treatment options.
New Formulation for HAE Approved
The FDA has approved a new oral pellet formulation of Orladeyo (berotralstat) for adults and adolescents 12 and older with hereditary angioedema (HAE).
HAE, is a rare genetic condition that causes sudden, unpredictable episodes of swelling in the skin, the gut, and sometimes the airway, which can be life-threatening.
Orladeyo is a once-daily prophylactic oral therapy that blocks plasma kallikrein to reduce the swelling that occurs in HAE. The original formulation, a capsule, was first approved in 2020 and the new pellet formulation was first approved for children aged 2 to 11 last December. The pellet formulation is now an for adolescents and adults who have trouble swallowing capsules or prefer the newer formulation.
From a business perspective, this is an example of lifecycle management in the pharmaceutical industry. Companies don’t always need to develop an entirely new drug to improve treatment. Sometimes, making an existing therapy easier to administer or more convenient for patients can deliver meaningful value.
FDA Approves Voceryq to Reduce Surgeries in Recurrent Respiratory Papillomatosis
On October 30th, the FDA granted accelerated approval to Voceryq from Inovio Pharmaceuticals to reduce the need for surgery in adults with recurrent respiratory papillomatosis (RRP).
RRP causes small, wart-like growths in the airway, most often on the vocal cords and in the larynx. It’s caused by HPV types 6 and 11 viruses. The growths aren’t cancerous, but they can change a person’s voice, and can potentially block the airway.
The standard treatment is surgery to remove the growths. The problem is the growths tend to return afterwards.
Voceryq contains two small circular pieces of DNA (plasmids). One plasmid carries instructions for HPV proteins, and the other for interleukin-12, to boost the immune response. In the trial that led to the accelerated approval, 26 patients had a median of 4.5 surgeries in the year before treatment. After treatment, 27% of patients reported not requiring any surgery for the next 12 months. The trial was relatively small and the FDA granted the drug an accelerated approval to allow patients access to a new treatment based on evidence that is reasonably likely to predict clinical benefit. However, the evidence is still preliminary and the developer (Inovio) needs to conduct a confirmatory trial to verify the benefit.
This is the second treatment approved for RRP (the first was Papzimeos). For physicians and patients, having another treatment option is important. It’s also a reminder that once a first therapy becomes available in a rare disease, it can create opportunities for additional innovation and competition.
3D Printed Treatment Options
Our last story focuses on PharmaTher, a company that has asked the FDA for orphan drug and rare pediatric disease designations for a 3D-printed form of creatine monohydrate to treat patients with called GAMT deficiency.
GAMT deficiency is an inherited metabolic disorder where the body can’t make creatine and toxins build up. If it isn’t treated, children can develop intellectual disability, seizures, speech problems, and movement disorders by age three.
Treatment is a strict diet that includes large doses of creatine and other supplements, given several times a day. And because these are supplements, insurance may not cover them. If this product gets approved as a prescription drug, the patient’s insurers would cover the cost, not the family.
From a practical perspective, 3D printing can offer a more flexible way to manufacture medicines in smaller quantities, with the ability to adjust doses as patients grow or their needs change. It’s still very early for PharmaTher’s proposed GAMT product. The company has submitted applications for designations, but the product hasn’t entered clinical trials.
References
New Formulation of Berotralstat Approved for Adults and Adolescents with Hereditary Angioedema
FDA Approves Berotralstat Oral Pellets for Adults and Adolescents with HAE (HCPLive)
https://www.hcplive.com/view/fda-approves-berotralstat-oral-pellets-adults-adolescents-hae
FDA Approves First Plasmid DNA-Based Immunotherapy for Adults with Recurrent Respiratory Papillomatosis
Individualized Human Genome Editing Products: Considerations for BLA Submission and Postapproval Management; Draft Guidance for Industry
FDA and CMS Announce New Steps to Advance Gene Therapies and Expand Access for Patients with Rare Diseases
https://www.hhs.gov/press-room/fda-cms-advance-gene-therapies-rare-diseases-patient-access.html
Commercial Performance of AAV Gene Therapies: Market Review and Future Implications (Recon Strategy)
PharmaTher Submits Orphan Drug and Rare Pediatric Disease Designation Applications to FDA for 3D-Printed Creatine Monohydrate in GAMT Deficiency
PharmaTher Launches M3DICINES National Network Program for 3D-Printed Medicines