Editors' (re)View: First treatment for rare bone disease; Mifepristone primed for SCOTUS showdown

Aug. 18, 2023
Pharma Manufacturing editors Karen Langhauser and Andrea Corona comment on the notable happenings in the pharma industry from the week of August 14

Editor’s note: Welcome to Editors' (re)View, our editors’ takes on things going on in the pharma world that deserve some extra consideration.

Strides in rare disease treatment

For patients living with fibrodysplasia ossificans progressiva (FOP), treatment options are limited. The genetic condition is progressive and causes abnormal bone growth that cannot be removed by surgery, and eventually impacts the ability to move and breathe. 

But earlier this week, the FDA approved the first treatment indicated for FOP, Ipsen's palovarotene, to be sold as Sohonos. The drug, administered as a capsule, belongs to a class of medicines known as retinoids, similar in mechanism to other drugs which are derivatives of vitamin A. The oral drug was first approved for use in Canada in January 2022, making it the first global approval for any FOP therapy. While it had received Rare Pediatric Disease and Breakthrough Therapy designations from the FDA, its road to approval in the U.S. was bumpy. 

Initially, Ipsen's NDA for the drug was accepted by the FDA in May 2021, but later withdrawn. After discussions with the agency, Ipsen needed to provide more data from its phase 3 MOVE and FOP programs. In December 2022, Ipsen received a Complete Response letter for the NDA. The 18-month trial with 107 participants ultimately exhibited a 54% reduction in abnormal bone growth volume compared to untreated individuals.

Although the drug comes at a hefty price — Ipsen has said that will be listed at $624,000 — for those living with FOP, Sohonos could be life-changing. 

Mifepristone primed for SCOTUS showdown

Earlier this week, a federal appeals court issued another ruling on the abortion drug, mifepristone. While the 5th Circuit Court of Appeals in New Orleans said that mifepristone should remain legal in the U.S., it ruled in favor of significant restrictions on patient access. If the ruling were to go into effect, it would revert to 2016 requirements for the use of mifepristone. 

In 2016, the FDA took steps to ease access restrictions for mifepristone. Those actions included a change in the drug's REMS program which increased the maximum gestational age to 70 days, eliminated the in-person dispensing requirement, allowed non-doctors to prescribe and administer mifepristone, and eliminated reporting of non-fatal adverse events. It's those 2016 amendments with which the appeals court has taken issue.

However, despite this recent ruling, nothing will actually happen at this point. Following the radical April ruling by a U.S. District Court Judge in Texas that suspended the FDA approval of mifepristone, SCOTUS weighed in, saying that mifepristone would have to remain available under the current rules until the appeals process played out.

So while nothing changes in terms of mifepristone access at the moment, the recent ruling has teed up an inevitable showdown before the Supreme Court — and a lot more than abortion drugs could get caught in the crossfire.

—  Karen Langhauser

About the Author

Andrea Corona | Senior Editor

About the Author

Karen P. Langhauser | Chief Content Director, Pharma Manufacturing

Karen currently serves as Pharma Manufacturing's chief content director.

Formerly the editor-in-chief of Food Manufacturing magazine, Karen was particularly successful at eating all of the snacks that were mailed to her from food companies, as well as reaching readers by establishing her own unique voice and tone on the brand. She made the decision to trade food for drugs in 2013, when she joined Putman Media as the digital content manager for Pharma Manufacturing.

As an award-winning journalist with 20+ years experience in the manufacturing space, Karen passionately believes that B2B content does not have to suck. As the content director, her ongoing mission has been to keep Pharma Manufacturing's editorial look, tone and content fresh and accessible.

Karen graduated with honors from Bucknell University, where she majored in English and played Division 1 softball for the Bison. Happily living in NJ's famed Asbury Park, Karen is a retired Garden State Rollergirl, known to the roller derby community as the 'Predator-in-Chief.'